A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

Surveys of caregivers and health care providers reveal gaps in screening and management of neuropsychiatric and neurodevelopmental symptoms among patients with Duchenne muscular dystrophy in the ...

A comprehensive care approach can help people with ambulatory DMD maximize function and retain the ability to walk for as ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular ...

Surveys of caregivers and health care providers reveal gaps in screening and management of neuropsychiatric and neurodevelopmental symptoms among patients with Duchenne muscular dystrophy in the ...

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

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

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

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

In 2023, Elevidys received expedited U.S. approval despite concerns from some Food and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the ...