The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

The beginning to the year has been eventful in the Duchenne muscular dystrophy (DMD ... who was dosed with Sarepta Therapeutic’s DMD gene therapy Elevidys (delandistrogene moxeparvovec ...

It’s the first known patient death with the therapy, which has been ... about its effectiveness in treating Duchenne muscular dystrophy. It’s the first gene therapy approved in the U.S ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young man died of acute ...