Researchers from University College London (UCL) are aiming to develop potential treatments for congenital muscular dystrophy ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the importance of a smooth transition from pediatric to adult care for patients with ...

Michael Rankin, 12, who is desperately waiting for access to the drug, said: “Every second they don’t get this through, one ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared updates ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

The Jaffa Shriners’ DuBois Area Shrine Club continues to support a wheelchair program that provides essential equipment to ...