Sarepta Therapeutics SRPT announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD) ...

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 ...

(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 ...

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 ...

QMUS may serve as a reliable, non-invasive biomarker for monitoring DMD progression and functional status, addressing limitations of traditional assessments. Strong correlations were found between ...

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 ...

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 ...

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 ...