The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...

Vinay Prasad, who came under fire from patient advocacy groups and Laura Loomer, is gone after less than three months on the ...

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

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...