Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Johnny Quintana’s life changed at the age of 19 when he was diagnosed with a rare form of muscular dystrophy called ...

As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...