One of the most common forms of muscular dystrophy is Duchenne muscular dystrophy (Duchenne). Globally, Duchenne is the most prevalent form of muscular dystrophy in children. It primarily affects boys ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

WASHINGTON, June 12, 2025 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is pleased to announce ...

Ifetroban improved heart function and reduced cardiac damage biomarkers in people with DMD in a Phase 2 clinical trial.

The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...

Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...

In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

Artificial intelligence (AI)–based electrocardiogram interpretation (AI-ECG) detected left ventricular systolic dysfunction ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...