Landmark Approval for a Life-Changing Treatment
Duchenne Muscular Dystrophy (DMD) Therapy
The Food and Drug Administration (FDA) announced its accelerated approval of SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) in 2023 following decades of research in the Center for Gene Therapy at Nationwide Children's Hospital 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 emeritus in the Center for Gene Therapy, 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.
Spinal Muscular Atrophy Type 1 (SMA1) Therapy
Following astonishing results of clinical research conducted here for a devastating genetic condition called spinal muscular atrophy type 1 (SMA1), the FDA issued an approval for a gene therapy that has changed the lives of children with SMA all over the world.
The therapy, made possible because of decades of research at Nationwide Children’s and The Ohio State University, has allowed children not only to live, but to crawl, walk and run. Jerry Mendell, MD, principal investigator emeritus in the Center for Gene Therapy at Nationwide Children's, conducted the first experimental trial for SMA type 1 here. In 2017, the readers of Science magazine named the results of this trial the most important scientific breakthrough of that year. The FDA approved the therapy, called Zolgensma, in 2019.
