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

Groundbreaking advancements in gene therapy at UTMB represent new possibilities for patients living with rare genetic ...

Mother charged after 12-year-old boy dies from starvation ...

A milestone in the treatment of Duchenne muscular dystrophy (DMD) passed recently with remarkably little public notice. That ...

For more than a year, a Wisconsin mother failed to care for her 12-year-old disabled son which led to his death weighing just ...

Three years after Elevidys treatment, boys with DMD show sustained motor function compared with expected disease progression.

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Researchers described what they believe to be the first case of SMA type 4 presenting with a false enlargement of a muscle in ...

Discusses 3-Year Topline Results From EMBARK Phase 3 Trial of ELEVIDYS in Duchenne Muscular Dystrophy January 26, ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products ...

Sarepta reports positive, statistically significant results in a study evaluating the efficacy of its blockbuster gene ...

Sarepta Therapeutics (NasdaqGS:SRPT) reported positive three year functional results from its Phase 3 EMBARK trial of Elevidys in ambulatory Duchenne muscular dystrophy patients. The data show slowed ...