Insmed’s (INSM) treatment of Duchenne muscular dystrophy, the single-stranded, non-replicating, recombinant adeno-associated virus serotype 9 ...

The first few months of 2025 brought hope to the rare disease community. Food and Drug Administration leadership promised ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

The FDA’s shift on Elevidys sends a message: boldness, scientific rigor and urgency are essential in confronting rare ...

Echocardiography may be at the vanguard for early and rapid detection of heart issues indicative of Duchenne muscular ...

A group of pharma companies and Duchenne UK are collaborating to improve the chances of new treatments for Duchenne muscular dystrophy gaining NICE approval. Roche, Pfizer, PTC Therapeutics, and ...

Sarepta Therapeutics obtained traditional FDA approval for ambulatory DMD patients and accelerated approval for ...

Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

This September, the Muscular Dystrophy Association (MDA) is launching its milestone #MDAstrong campaign during a historic moment for the organization: its 75th anniversary. The campaign reframes what ...