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 ...

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

MyHealthTeam, a Swoop company, the creator of one of the largest, engaged patient social networks in healthcare, has announced the launch of myMDteam. This new website and patient community is ...

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

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

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

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

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...