JerryRMendellMD

Interventional Neurology, Neuromuscular Medicine, Child Neurology

Professor of Pediatrics and Neurology, Ohio State University Curran Peters Chair of Research Translational Gene Therapy Scientist

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Office
700 Childrens Dr
Columbus, OH 43205
Phone+1 614-355-3574

Fax+1 614-722-3273

Education & Training

Clinical Center at the National Institutes of HealthFellowship, Neuromuscular Medicine (Neurology), 1969 - 1972
New York Presbyterian Hospital (Columbia Campus)Residency, Neurology, 1966 - 1969
University of Texas Southwestern Medical SchoolClass of 1966

Certifications & Licensure

OH State Medical License 1972 - 2024
TX State Medical License 1966 - 1988
American Board of Psychiatry and Neurology Neurology

Awards, Honors, & Recognition

Sidney Carter Award in Child Neurology American Academy of Neurology 2021
America's Top Doctors Castle Connolly, 2002-2022
Bernard Sachs Lecture Child Neurology Society, 2021
National Academy of Medicine 2020
Allen Distinguished Scholar Award Nationwide Childrens Hospital, 2019
Lifetime Achievement Award Limb Girdle Muscular Dystrophy Foundation, 2019
Clinical Research Forum Distinguished Achievement National Press Club Washington DC, 2018
Science Magazine Breakthrough Achievement Award Science Magazine, 2017
Distinguished Scholar Ohio State University 2009
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Clinical Trials

Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease Start of enrollment: 1992 Oct 01
Completed
Phase 2
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Intravenous Immune Globulin for Chronic Inflammatory Demyelinating Polyneuropathy Start of enrollment: 1992 Sep 01
Completed
Phase 3
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders Start of enrollment: 1992 Jun 01
Completed
Phase 3
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy Start of enrollment: 2006 Mar 01
Completed
Phase 1
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons Start of enrollment: 2007 Mar 01
Completed
Phase 1
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy Start of enrollment: 2005 Jun 01
Completed
Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) Start of enrollment: 2008 Mar 01
Completed
Phase 1
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients Start of enrollment: 2011 Jul 01
Completed
Phase 2
Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis Start of enrollment: 2012 Jan 01
Completed
Phase 1
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Start of enrollment: 2012 Feb 27
Completed
Phase 2
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Start of enrollment: 2014 May 05
Completed
Phase 1
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy Start of enrollment: 2014 Apr 17
Completed
Phase 2
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy Start of enrollment: 2015 Jun 30
Completed
Phase 2
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) Start of enrollment: 2018 Jan 04
Completed
Phase 1, Phase 2
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study Start of enrollment: 2018 Mar 28
Recruiting
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Publications & Presentations

PubMed

223 citations
Disruption of DAG1 in differentiated skeletal muscle reveals a role for dystroglycan in muscle regeneration.
Ronald D. Cohn, Michael D. Henry, Daniel E. Michele, Rita Barresi, Fumiaki Saito
Cell. 2002-09-06
1311 citations
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell, W. Dave Arnold, Louise R. Rodino-Klapac
The New England Journal of Medicine. 2017-11-01
359 citations
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Jerry R. Mendell, Samiah Al-Zaidy, Louise R. Rodino-Klapac, Kimberly Goodspeed, Steven J. Gray
Molecular Therapy. 2021-02-03

Journal Articles

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children with Duchenne Muscular Dystrophy
Jerry R Mendell, Zarife Sahenk, Samiah Al-Zaidy, Richard Shell, JAMA Neurology

Authored Content

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children with Duchenne Muscular DystrophyJune 2020

Press Mentions

Gene Therapy Details Revealed in Duchenne TrialOctober 18th, 2024
Nationwide Children's Names Gene Therapy Center for Retired Pioneer Dr. Jerry MendellSeptember 13th, 2024
MD Gene Therapy Invented at Nationwide Children's Could Hit $4B Annual Sales if ApprovedJanuary 31st, 2023
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Grant Support

Vascular Delivery Of Alpha-Sarcoglycan For Lgmd2dNational Institute Of Arthritis And Musculoskeletal And Skin Diseases2011
The Implications Of Dystrophin-Specific T Cells For DMD Gene CorrectionEunice Kennedy Shriver National Institute Of Child Health &Human Development2010–2011
Overcoming Immune Barriers To Gene Correction For Duchenne Muscular DystrophyEunice Kennedy Shriver National Institute Of Child Health &Human Development2010–2011
Administrative CoreEunice Kennedy Shriver National Institute Of Child Health &Human Development2010–2011
Dystrophin RestorationNational Institute Of Neurological Disorders And Stroke2007–2010
Diverse Strategies To Correct The Dystrophin Gene Using Vascular Delivery (U54)National Institute Of Neurological Disorders And Stroke2007–2010
Implementing Newborn Screening For Duchenne Muscular Dystrophy In The CommunityCenters For Disease Control And Prevention2007–2009
Clinical Trial: Six-Month (26 Weeks) Clinical Trial Of Gentamicin In Duchenne MDNational Center For Research Resources2008
Developing Clinical Outcomes For Gene TransferNational Institute Of Arthritis And Musculoskeletal And Skin Diseases2003–2008
Administrative CoreNational Institute Of Neurological Disorders And Stroke2007
Study Of SR 57746a In Patients With Amyotrophic Lateral SclerosisNational Center For Research Resources2007
Study Of MYO-029National Center For Research Resources2007
Six-Month (26 Weeks) Clinical Trial Of Gentamicin In Duchenne MDNational Center For Research Resources2006–2007
Newborn Screening For Duchenne Muscular DystrophyCenters For Disease Control And Prevention2004–2007
Gentamicin Trial In Duchenne And Limb Girdle DystrophiesNational Institute Of Neurological Disorders And Stroke2004
Gentamicin Trial In Duchenne And Limb Girdle DystrophiesNational Institute Of Neurological Disorders And Stroke2002–2003
LGMD Study GroupNational Center For Research Resources1999–2001
Great Lakes ALS Study GroupNational Center For Research Resources1999–2001
Efficacy, Safety, Tolerability Of Sr57746a In ALS PatientsNational Center For Research Resources1999–2001
Efficacy Of Sr57746a In Patients With Amyotrophic Lateral SclerosisNational Center For Research Resources1998–2001
Clinical Trial Of Oxandrin In Treatment Of Duchennes Muscular DystrophyNational Center For Research Resources1998–2001
Intravenous Immunoglobulin In CipdNational Center For Research Resources1996–2001
Treatment Of Patients With Amyotrophic Lateral Sclerosis Using A Ketogenic DietNational Center For Research Resources2000
Safety, Efficacy And Tolerability Of Beta Interferon1a In Inclusion Body MyositisNational Center For Research Resources2000
Multicenter Trial In Periodic Paralysis And Sodium Channel DisorderNational Center For Research Resources1997
Androgen Hormone Deficiency And Replacement In Motor Neuron DiseaseNational Center For Research Resources1996–1997
Myoblast Transfer Studies In Duchenne Muscular DystrophyNational Center For Research Resources1996
Multicenter Trial In Periodic Paralysis &Associated Sodium Channel DisorderNational Center For Research Resources1996