Sarepta Therapeutics (NasdaqGS:SRPT) reported positive three year functional results from its Phase 3 EMBARK trial of ...

Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For ...

The FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys), the first gene therapy for Duchenne muscular dystrophy, the agency announced Thursday. The adeno-associated virus-based ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

Disability advocate TaLisha Gryzb is a mother of four living with muscular dystrophy who educates viewers about her ...