FDA Approves First Gene Therapy Treatment for Duchenne Muscular Dystrophy Developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital
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The Abigail Wexner Research Institute at Nationwide Children’s Hospital praised the Food and Drug Administration (FDA) for its accelerated approval of SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in its Center for Gene Therapy to help patients with neuromuscular diseases.
The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute, led the first Phase I study of a systemic gene therapy for DMD. Dr. Mendell saw his first patient with DMD more than 50 years ago, igniting his passion to develop new treatments for patients with neuromuscular diseases.
