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 ...

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

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

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 ...

A team from UC San Diego, John Hopkins and UC Berkeley Universities found a way to edit a single gene in a mosquito that ...

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

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

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

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

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