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

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

What has Wall Street been buzzing about this week? Here are the top 5 Buy calls and the top 5 Sell calls made by Wall Street’s best analysts ...

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

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

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

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

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

Sarepta Therapeutics' brutal week continued Friday after European officials rejected the company's controversial gene therapy ...

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

The FDA is still on track to meet its user fee targets despite a higher-than-normal vacancy rate among scientific reviewers.