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

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 ...

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

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

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

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

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 company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

Sarepta Therapeutics (SRPT) said late Monday it will pause all shipments of its Duchenne muscular dystrophy gene therapy, ...

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