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

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

Europe's medicines regulator did not recommend the approval of Sarepta Therapeutics' gene therapy Elevidys for a type of rare ...

Roche Holding AG’s gene therapy Elevidys failed to get the backing of European regulators, a blow to the Swiss drugmaker and to Sarepta Therapeutics Inc., the embattled US biotech that developed the ...

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

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

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

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

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...

Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...

Friday said that the European Medicines Agency has issued a negative opinion on the conditional marketing authorization for Elevidys, intended for ambulatory patients aged three to seven with Duchenne ...

Over 5 years of treatment in hemophilia B patients, gene therapy research shows sustained factor IX activity and safety.