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

Families say it's 'completely frustrating' that children with Duchenne Muscular Dystrophy are being denied access to free ...

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).

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

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

Dozens of families affected by Duchenne muscular dystrophy (DMD) have urged the Scottish Government to ensure a life-saving ...

Duchenne muscular dystrophy (DMD) patients in Scotland have not been able to get the drug – which is available in England – despite manufacturers offering it to health boards for free on an ...

Key treatments for Alzheimer’s, rare cancers, and infectious diseases are under review, with new vaccines and therapies like ...