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

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

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

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

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

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.

After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...