Marc Bigger was just 19-years-old when he passed away from the muscle-wasting condition, Duchenne Muscular Dystrophy, in ...

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

A Champaign second-grader hopes her family's fundraiser can help a kid in the future have a more "normal" childhood, not ...

The Health Resources and Services Administration is requesting public comments on its recommendation to add metachromatic ...

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...

After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be ...

A well-connected drug company and Laura Loomer wanted Kennedy ally Vinay Prasad gone. Trump Chief of Staff Susie Wiles got ...

Dr Essajee says that in Kenya, treatment is largely supportive: “Steroids like Deflazacort, combined with vitamin D and ...

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular ...

Anaheim Ducks right wing Troy Terry is trading his hockey stick for a driver this September as he takes on a new kind of role ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

Pratteln, Switzerland, 13 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Gen Ilaç ve Saglik Ürünleri San. ve Tic. A.S. (GEN) for the ...