Trusted Resources: People & Places
Healthcare providers, researchers, and advocates
Jerry R. Mendell, MD
Researcher Attending Neurologist
Pediatrics & Neurology
Nationwide Children’s Hospital
700 Children's Drive
Columbus, Ohio, United States
Jerry R. Mendell, MD, is an Attending Neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at Nationwide Children’s, and The Ohio State University.
He is a Clinician Scientist with decades of experience. Dr. Jerry R. Mendell has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the Principal Investigator on the Prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.
Representative Publications:
Health Outcomes in Spinal Muscular Atrophy Type 1 Following AVXS-101 Gene Replacement Therapy
Related Content
-
SMA Gene Replacement Therapyhttps://www.youtube.com/watch?v=vHbJs8bm...
-
Gene Replacement Therapy With Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy Aged 24 Months or Yo...Given the novelty of gene replacement th...
-
Lee Rubin, PhDLee Rubin investigates the key molecular...
-
Coverage of Genetic Therapies for Spinal Muscular Atrophy Across Fee-for-Service Medicaid ProgramsBackground: Genetic therapies are a prom...
-
Kathie Bishop, PhDKathie Bishop is a Ph.D level neuroscien...
-
Jennifer M. Kwon, MDJennifer M. Kwon, MD is board certified ...
-
Crystal Proud, MD, PhDDr. Proud is a board-certified pediatric...