Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

Commercial launch in Switzerland anticipated in H2 2026Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

Together with his brothers, twin Edward, younger sibling William, 24, big brother Oliver, 30, and their cousin Rupert Lowther ...

"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...

Mary Wickizer, center, shows her son Isaac, 7, the keys to their new wheelchair-accessible van while her husband Matt Wickizer looks on during a special presentation on Nov. 22 at Superior Van & ...

PPMD extends its heartfelt thanks to all Race to End Duchenne runners, donors, and volunteers who made the 2026 Walt Disney World (R) Marathon Weekend a memorable and meaningful success. To learn more ...

Tributes flooded the internet for Gilbert Gottfried after his family shared on Tuesday via Twitter that he died following “a long illness.” Since then, there has been more awareness around muscular ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...