A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found. Altruism values for ...

Therapeutics are down 24.95, or 25%, to $76.40 in pre-market trading after the company shared a safety update related ...