FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy 1024 632 Abbie Miller

On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital.

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.

“This is what I’ve devoted my life to; since I began investigating gene therapy as a potential treatment for children with neuromuscular disorders, it’s been my dream to develop a gene therapy for DMD,” Dr. Mendell said. “The FDA’s decision means we can save more function, and save the quality of life for these children and their families.”


Read the April 2023 feature story about micro-dystrophin for DMD.



Image credit: Mandy Root-Thompson for Nationwide Children’s

Video: Nationwide Children’s

About the author

Abbie (Roth) Miller, MWC, is a passionate communicator of science. As the manager, medical and science content, at Nationwide Children’s Hospital, she shares stories about innovative research and discovery with audiences ranging from parents to preeminent researchers and leaders. Before coming to Nationwide Children’s, Abbie used her communication skills to engage audiences with a wide variety of science topics. She is a Medical Writer Certified®, credentialed by the American Medical Writers Association.