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 The Ohio State University.

Dr. Mendell joined the Research Institute fulltime in 2004, recruited by Philip Johnson, MD, the director of the Center for Gene Therapy at that time. Following Dr. Johnson’s departure, Dr. Mendell became the center’s director until 2017 when he began to fully devote his time toward his many gene-based therapy research initiatives at Nationwide Children’s.

Dr. Mendell is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside.
He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He 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 has been the principal investigator on numerous clinical trials including the prednisone clinical trial for DMD in 1989 that has profoundly influenced clinical care; the exon-skipping trial using eteplirsen, which the FDA approved for clinical use in 2016; the first gene therapy trials for DMD and LGMD; and the SMA gene therapy trial that was the first systemically delivered gene therapy approved by the FDA in 2019 specifically for infants with SMA type 1.