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.

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

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

Delandistrogene moxeparvovec showed significant long-term stabilization or slowed progression in DMD patients over 3 and 5 years compared to external controls. Clinical trials demonstrated ...

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

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 ...

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

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 ...

Taiho Pharmaceutical’s investigational treatment for Duchenne muscular dystrophy (DMD) has failed to impact the time it takes patients to rise from the floor over 52 weeks, missing the phase 3 study’s ...

Following a review of safety data in the ambulatory DMD population, the FDA had recommended the removal of the voluntary hold for these patients. The Food and Drug Administration (FDA) is allowing ...

Credit: Cranbury Pharmaceuticals. The generic product is being made available by Cranbury Pharmaceuticals. The generic product labeling only includes information for DMD patients 5 years of age and ...