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 ...

A teenager living with a severe and degenerative condition is hoping to retain his independence in the face of the cruel ...

The Duchenne muscular dystrophy market is witnessing steady growth driven by advancements in gene therapies, exon-skipping drugs, and corticosteroid alternatives. Increasing diagnosis rates, rising ...

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

Webinar to be held on Tuesday, July 29, 2025, at 1:00 p.m. ET – SAN DIEGO, July 28, 2025 (GLOBE NEWSWIRE) -- Capricor ...

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

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

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 ...