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

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

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

Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his handling of Sarepta Therapeutics Inc.’s gene therapy. “Dr. Prasad did not want to ...

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

In its own communique issued shortly after the FDA’s, Sarepta elaborated on the situation and confirmed a Roche statement to ...