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

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

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.

Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

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 (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...

Sarepta has suffered another regulatory setback after the European Medicines Agency’s (EMA’s) Committee for Medicinal ...

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

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

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