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 ...

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

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

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

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

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

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

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

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...

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

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...