Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

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 ...

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 ...

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 ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...

Solid Biosciences (SLDB) stock rises as a Phase 1/2 trial for its lead asset SGT-003 in Duchenne muscular dystrophy reaches ...

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) ...

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 & ...

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 ...

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