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 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.

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 ...

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

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

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

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 ...

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

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

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