An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

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

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

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

The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...

FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...

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

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

Quantitative muscle ultrasound correlates strongly with ambulatory and timed function tests in Duchenne muscular dystrophy, ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

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