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 ...

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

Dr. Michio Hirano to receive the MDA Legacy Award for Achievement in Clinical Research; Allison Moore to receive the MDA ...

Shares of  Regenxbio rose Monday in premarket trading after the company said its treatment for Duchenne muscular dystrophy delivered positive trial results, strengthening the case for a speedier ...

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 ...

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 ...

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 ...

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

Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

The FDA approved oral givinostat (Duvyzat) to treat Duchenne muscular dystrophy (DMD) in patients ages 6 years and older, the agency announced Thursday. Givinostat is a histone deacetylase (HDAC) ...

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 ...