In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

(Reuters) -The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...

Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...

Sarepta Therapeutics said it will resume shipments of its gene therapy Elevidys® (delandistrogene moxeparvovec-rokl) “imminently” to ambulant patients with Duchenne muscular dystrophy (DMD), ending a ...

Sarepta Therapeutics Inc. shares rose sharply early Tuesday after US regulators reversed course and recommended that patients ...

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its ...