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

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

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

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

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

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

Sarepta Therapeutics (SRPT) in focus as the biotech says the death of a patient who had received its gene therapy Elevidys ...

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

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...