F.D.A. Sharply Limits Use of Drug Linked to Two Teen Deaths

November 14, 2025

picture of the U.S. Food and Drug Administration sign

(New York Times) – A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients.

The Food and Drug Administration said on Friday that it would narrow approval of a gene therapy treatment for young men with a muscle-wasting disease.

The agency’s decision formalized an agreement reached with the drugmaker Sarepta in July to stop shipping it to certain patients after two teenagers using the medication died from liver failure, a known complication.

The agency said it was limiting the approved use of Elevidys after a safety review confirmed that the teenagers suffered liver damage that led to their hospitalizations and deaths. (Read More)