Linda Pax Lowes

Contact Information

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Pax Lowes, PT, PhD

Center for Gene Therapy700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Linda Pax Lowes

Biography

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

Awards, Honors & Organizations

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

Research

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

View More Publications

Education

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Professional Experience

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

Contact Information

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Pax Lowes, PT, PhD

Center for Gene Therapy700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Linda Pax Lowes

Biography

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

Awards, Honors & Organizations

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

Research

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

View More Publications

Education

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Professional Experience

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

Contact Information

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Pax Lowes, PT, PhD

Center for Gene Therapy700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Linda Pax Lowes

Contact Information

• Call us at:
• (614) 722-6881
• Fax us at:
• (614) 355-5247
• Email Linda Pax Lowes, PT, PhD
• Center for Gene Therapy700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Linda Pax Lowes

Biography

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Biography

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Biography

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases. The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft. In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Dr. Linda Lowes, a physical therapist and researcher specializing in the assessment of neuromuscular dystrophies, is a principal investigator in the Center for Gene Therapy and a research associate professor of Pediatrics at The Ohio State University. She directs the Lowes Neuromuscular Outcome Measures Lab, where she and her team develop new methods for measuring function and movement in people with neuromuscular diseases.

The Lowes Lab specializes in identifying outcome measures for rare diseases and neuromuscular disorders to better guide the development and assessment of new therapies in clinical trials. The team also develops innovative new measurement methods, including the ACTIVE Seated and the ACTIVE Mini. These systems are designed to assess movement in newborns using video game technology and are currently being tested for wide-scale use in partnership with Microsoft.

In 2019 the Lowes Lab was named the Outstanding Team of the Year at the Abigail Wexner Research Institute. Dr. Lowes has published more than 50 articles.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

Center for Gene Therapy

Principal Investigator

Lowes Lab

Principal Investigator

Primary Department

Center for Gene Therapy

• Center for Gene Therapy
• Principal Investigator
• Lowes Lab
• Principal Investigator
• Primary Department
• Center for Gene Therapy

Awards, Honors & Organizations

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

Awards, Honors & Organizations

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

Awards, Honors & Organizations

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

Outstanding Team of the Year, Research Institute, 2019 Excellence in Innovation Award, Research Institute, 2019 Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019 Executive Board, TREAT-NMD, 2019 - Present Member, TACT Advisory Board, 2015 - Present Member, World Muscle Association, 2010 - Present Member, TREAT-NMD, 2005 - Present Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001 Member, American Physical Therapy Association, 1983 - Present

• Outstanding Team of the Year, Research Institute, 2019
• Excellence in Innovation Award, Research Institute, 2019
• Appointed to the Ohio Occupational Therapy, Physical Therapy and Athletic Trainers Board, 2019
• Executive Board, TREAT-NMD, 2019 - Present
• Member, TACT Advisory Board, 2015 - Present
• Member, World Muscle Association, 2010 - Present
• Member, TREAT-NMD, 2005 - Present
• Pediatric Clinical Specialist, American Physical Therapy Association, 1996 - 2001
• Member, American Physical Therapy Association, 1983 - Present

Research

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

View More Publications

Research

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

View More Publications

Research

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

View More Publications

Lab(s)

Center for Gene Therapy

View My Publications Publications

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  

                


                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.

                


                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.

                


                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.

                


                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  

                


                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.

                


                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.

                


                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.

                


                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.

                


                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.

                


                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.

                


                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.

                


                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.

                


                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.

                


                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.

                


                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.

                


                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  

                


                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

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Lab(s)

Center for Gene Therapy

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Lab(s)

Center for Gene Therapy

• Center for Gene Therapy

                  Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;  
  
  
  
                  Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.
  
  
  
                  Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.
  
  
  
                  Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.
  
  
  
                  Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;  
  
  
  
                  Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.
  
  
  
                  Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.
  
  
  
                  Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.
  
  
  
                  Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.
  
  
  
                  Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.
  
  
  
                  Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.
  
  
  
                  Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.
  
  
  
                  James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.
  
  
  
                  Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.
  
  
  
                  Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.
  
  
  
                  Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.
  
  
  
                  Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.
  
  
  
                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  
  
  
  
                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.
  
  
  
                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.
  
  
  
                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  
  
  
  
                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  
  
  
  
                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.
  
  
  
                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.
  
  
  
                  Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;  
  
  
  
                  Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.
  
  

View More Publications

• Moore U, Fernandez-Torron R, Jacobs M, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Harris E, Guglieri M, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Jain COS Consortium., Bourke J, Straub V. Cardiac and pulmonary findings in dysferlinopathy: a 3-year, longitudinal study. Muscle Nerve. 2022 Feb 18;
• Gauthier LV, Nichols-Larsen DS, Uswatte G, Strahl N, Simeo M, Proffitt R, Kelly K, Crawfis R, Taub E, Morris D, Lowes LP, Mark V, Borstad A. Video game rehabilitation for outpatient stroke (VIGoROUS): A multi-site randomized controlled trial of in-home, self-managed, upper-extremity therapy. EClinicalMedicine. 2022 Jan; 43: 101239.
• Mayhew AG, James MK, Moore U, Sutherland H, Jacobs M, Feng J, Lowes LP, Alfano LN, Muni Lofra R, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sánchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Gordish-Dressman H, Hilsden H, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Straub V. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13: 828525.
• Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy. PLoS One. 2022; 17: e0266845.
• Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2021 Nov 13;
• Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Validity and Reliability of the Neuromuscular Gross Motor Outcome. Pediatr Neurol. 2021 Sep; 122: 21-26.
• Contesse MG, Sapp ATL, Apkon SD, Lowes LP, Dalle Pazze L, Leffler MG. Reliability and construct validity of the Duchenne Video Assessment. Muscle Nerve. 2021 Aug; 64: 180-189.
• Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1; 78: 834-841.
• Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR, Jain COS Consortium., Mayhew AG, Straub V. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 May; 89: 967-978.
• Servais L, Camino E, Clement A, McDonald CM, Lukawy J, Lowes LP, Eggenspieler D, Cerreta F, Strijbos P. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases. Digit Biomark. 2021 May-Aug; 5: 183-190.
• Moore U, Gordish H, Diaz-Manera J, James MK, Mayhew AG, Guglieri M, Fernandez-Torron R, Rufibach LE, Feng J, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Lowes LP, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Apr; 31: 265-280.
• Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L, NatHis-SMA study group.. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann Clin Transl Neurol. 2021 Feb; 8: 359-373.
• James MK, Rose K, Alfano LN, Reash NF, Eagle M, Lowes LP. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic. Front Genet. 2021; 12: 735538.
• Duong T, Krosschell KJ, James MK, Nelson L, Alfano LN, Eichinger K, Mazzone E, Rose K, Lowes LP, Mayhew A, Florence J, King W, Senesac CR, Eagle M. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases. Front Genet. 2021; 12: 735936.
• Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, Eteplirsen Study Group.. Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls. J Neuromuscul Dis. 2021; 8: 469-479.
• Duong T, Harding G, Mannix S, Abel C, Phillips D, Alfano LN, Bönnemann CG, Lilien C, Lowes LP, Servais L, Warken-Madelung B, Nieto Bergman S, James ES, Noursalehi M, Prasad S, Rico S, Bilder DA. Use of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance. J Neuromuscul Dis. 2021; 8: 63-77.
• Alfano LN, Miller NF, Iammarino MA, Moore Clingenpeel M, Lowes SL, Dugan ME, Kissel JT, Al Zaidy S, Tsao CY, Lowes LP. ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol. 2020 Mar; 62: 303-309.
• Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L’Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;
• Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.
• Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.
• Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;
• Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;
• Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.
• Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.
• Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M, Ashida A, Sakamoto C, Tateishi T, Yajima H, Canal A, Ollivier G, Decostre V, Mendez JB, Sánchez-Aguilera Praxedes N, Thiele S, Siener C, Shierbecker J, Florence JM, Vandevelde B, DeWolf B, Hutchence M, Gee R, Prügel J, Maron E, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Díaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Assessment of disease progression in dysferlinopathy: A 1-year cohort study. Neurology. 2019 Jan 9;
• Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L, NatHis-SMA study group.. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018; 13: e0201004.

Education

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Education

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Education

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Doctorate

Hahnemann University Hospital

Date Completed: 06/30/1987

Undergraduate School

The Ohio State University

Date Completed: 06/30/1983

Professional Experience

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

Professional Experience

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

Professional Experience

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics2018 - Present Director of Lowes Neuromuscular Outcome Measures Lab, Principal Investigator2018 - Present The Ohio State University, Associate Professor, College of Medicine2015 - Present The Ohio State University, Clinical Assistant Professor, Neurology2000 - Present The Ohio State University, Adjunct Assistant Professor, School of Health and Rehabilitative Services

2018 - Present The Ohio State University, Associate Professor, Department of Pediatrics

Contact Information

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Center for Gene Therapy

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

Call us at: (614) 722-6881

Fax us at: (614) 355-5247

Email Linda Lowes

                    700 Children's DriveColumbus, OH 43205 (map)

• Call us at:
• (614) 722-6881
• Fax us at:
• (614) 355-5247
• Email Linda Lowes
• 700 Children’s DriveColumbus, OH 43205 (map)