A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Susan and Chris Finazzo have enrolled their sons Dylan and Chase in a study of gene therapy for Duchenne muscular dystrophy. The experimental treatment is still being studied but researchers hope it ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...

The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...