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

Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study With a Prospective Natural History Cohort

Share

Discuss in Social Wall

Share to rareTeam

Bookmark