SRPT jumps nearly 8% after Elevidys shows sustained motor gains and slower progression three years post-dose in the phase III EMBARK DMD study.

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

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 ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

William Blair analysts view Elevidys’ three-year outcome as a win, though remain sceptical on the readout’s impact on sales ...

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 ...

"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 ...

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) causes muscle weakness that becomes more severe over time. While there is no cure, treatments can help slow the progression and manage symptoms and complications.

(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 ...

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 ...