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

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 - WALTHAM, Mass., Aug. 04, 2025 ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

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

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

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

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

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...

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

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

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