💨 Abstract

Sarepta Therapeutics has defied the FDA's request to halt sales of its gene therapy, Elevidys, following three patient deaths. The first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, Elevidys is now in doubt. The FDA usually relies on informal requests, giving companies time to comply, but Sarepta's refusal has raised stakes. The company blames patients' liver injuries but insists the drug is safe for younger patients.

Courtesy: WTOP Staff