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 ...

Keros Therapeutics has shaken up its leadership structure as the bi | Keros has shaken up its leadership structure as the ...

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the second quarter of 2025 and recent business highlights.

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

K.S.R.A. Praneeth, an 18-year-old battling Duchenne Muscular Dystrophy, competed in the World IPCA Chess Championship in Goa, ...

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.

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

Swiss pharmaceutical giant Novartis is exploring a potential acquisition of U.S. biotech firm Avidity Biosciences, according ...

If a deal is confirmed and consummated, Novartis would claim ownership of a biotech that has raised something of a stir in ...

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