This year’s top content in the Duchenne muscular dystrophy (DMD) space focused on a novel gene therapy approval, the high cost of care for this genetic disorder, and steps forward in other treatments.

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Comparable long-term effectiveness to standard of care corticosteroids over 5 years Normal growth maintained, in contrast to growth suppression ...

Wave Life Sciences has met its goal in a Duchenne muscular dystrophy (DMD) study, positioning it to talk to regulators about accelerated approval while continuing to track patients through to the ...

The patient had DMD diagnosed when he was 5.5 years of age and had been treated over the course of 21 years with daily deflazacort (1.1 mg per kilogram per day). He lost the ability to ambulate ...

SAN ANTONIO — A family out of New Braunfels is in need of an accessible van to help support three brothers with Duchenne Muscular Dystrophy (DMD). Two of the kids rely on wheelchairs while the other ...

Cardiomyopathy is the leading cause of mortality in patients with DMD. Compared with their healthy counterparts, patients with DMD — including those with preserved left ventricular ejection fraction ...

Swiss specialty drugmaker Santhera Pharmaceuticals saw its shares rise 5.8% to 10.90 francs today, after it reported positive ...

Sarepta’s stock plunged about 38% in after-hours trading, after it closed the regular session up 1.8%. That decline would put ...

Sarepta Therapeutics, Inc. SRPT reported third-quarter 2025 adjusted loss of 13 cents per share against the Zacks Consensus ...