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

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 SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

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

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

Muscular Dystrophy (MD) is more than a medical condition—it's a journey that reshapes lives. Imagine trying to climb stairs, run, or even stand, only to find your muscles not cooperating. That’s the ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

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

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

The quest for a treatment for muscular dystrophy has led some scientists to focus on the half-dozen muscles surrounding the eyes. These extraocular muscles, which control eye movement, remain ...

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

US FDA grants Orphan Drug Designation to Atossa Therapeutics’ (Z)-endoxifen to treat Duchenne muscular dystrophy: Seattle Monday, January 19, 2026, 17:00 Hrs [IST] Atossa Therap ...