Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO announced the publication of preclinical results for its investigational gene ...

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

The FDA greenlit multiple new drugs this month and issued some notable label expansions including for Eli Lilly’s Kisunla.

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 ...

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

Donald Trump reportedly ignored calls from Health Secretary Robert F. Kennedy Jr. not to fire the top vaccine official at the Food and Drug Administration after a pressure campaign from far-right ...

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 ...

A senior official at the US Food and Drug Administration has suddenly resigned after only three months in the job. Vinay Prasad’s departure followed controversy over a treatment for Duchenne muscular ...