The pedigree demonstrated expression of ... abnormalities observed were similar to those observed in the Duchenne type of muscular dystrophy but differed in that variation in fiber size was ...

Regenxbio reported Monday the first clinical evidence showing improved muscle function in boys with Duchenne muscular dystrophy following treatment with its experimental gene therapy. The positive ...

The EMBARK Phase 3 study for Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, was published this month in Nature Medicine. The paper largely reiterates what’s ...