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

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

CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...

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

The European Medicines Agency’s drug advisory committee (CHMP) rejected Elevidys for children aged 3 to 7 years who are able ...

Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

This week's episode covers news about Sarepta, babies born following mitochondrial transfer, and AI identifying therapy ...

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

Sarepta said it accepts the CHMP decision on Elevidys, while partner Roche said it will continue working with the EMA.

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

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

The Food and Drug Administration won’t sign off on Sarepta Therapeutics Inc. bringing its controversial gene therapy back to ...