Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular ...

With a strong launch underway for a bladder cancer gene therapy, Ferring Pharmaceuticals is finding the kind of commercial ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The fate of Sarpeta’s gene therapy for the disease, called Elevidys, has been ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

European regulators on Friday said that a Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment that is facing increasing scrutiny in the ...

LOS ANGELES (KABC) -- A first-of-its-kind gene therapy was just approved to treat Duchenne muscular dystrophy, a devastating condition affecting roughly 1 out of 3,500 to 5,000 boys. The game ...

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 ...

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

A new paper, published in Gene Therapy, raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant ...

Second patient death reported with gene therapy for muscular dystrophy The U.S. Food and Drug Administration campus in Silver Spring, Md., is photographed, Oct. 14, 2015. Credit: AP/Andrew Harnik ...