WASHINGTON, D.C. — Federal health regulators are investigating the death of an 8-year-old boy who received Elevidys, a gene therapy developed by Sarepta Therapeutics for Duchenne muscular dystrophy, a rare and progressive genetic disorder.
The U.S. Food and Drug Administration confirmed the fatality occurred on June 7, 2025, and has requested a voluntary pause in the distribution of the therapy as it conducts a thorough safety review. Sarepta has complied with the FDA’s request.
Elevidys is a single-dose gene therapy delivered via intravenous infusion and is based on Sarepta’s AAVrh74 platform, an adeno-associated virus vector technology designed to deliver genetic material to muscle cells. The therapy was developed to address the underlying genetic defect responsible for Duchenne muscular dystrophy, a condition that leads to severe muscle degeneration and loss of mobility, primarily affecting boys.
The FDA has not yet released details on the circumstances of the death or whether a causal link has been established between the treatment and the incident. The investigation remains ongoing.
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