Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

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

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...