Viltolarsen targets dystrophin gene mutations, enabling functional dystrophin protein production, crucial for muscle health in patients with DMD. Consistent viltolarsen treatment slowed disease ...

Sarepta Therapeutics SRPT announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating ...

Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...

Givinostat, combined with corticosteroids, delays DMD progression and improves key mobility aspects in patients aged 6 and older. The study included 142 patients on givinostat and a matched natural ...

(RTTNews) - Capricor Therapeutics, Inc. (CAPR), on Monday, announced positive data from its ongoing HOPE-2 open-label extension trial, demonstrating the long-term efficacy of Deramiocel for the ...

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 ...

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up ...

"While we did see correlations between activity levels and cardiomyopathy progression in patients with Duchenne muscular dystrophy, these correlations were not particularly strong. Our interpretation ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Early screening for Duchenne muscular dystrophy can help you get early treatments and referrals for your child to help prevent further loss of muscle tissue. Duchenne muscular dystrophy (DMD) is a ...

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and ...