News

SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in DMD — and preserve muscle health for boys in a clinical trial, according to new data. The one-time treatment was also well…

PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting that the treatment is affecting its intended molecular target.  Based on these early…

Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function over several years of follow-up, new data showed. That stands in contrast to the typical progression of BMD, in which motor function steadily declines as the disease progresses. The data…

At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Muscular Dystrophy News Today sat down with John F. Crowley, president and CEO of the…

Eating more protein was associated with better lower limb function and a higher quality of life among people with muscular dystrophy (MD), regardless of their ability to walk, according to a recent analysis. However, in general, MD patients were not consuming protein at levels considered sufficient to stimulate muscle…

A new 3D model of Duchenne muscular dystrophy (DMD) reveals that while gene therapy can bolster muscle strength, it may be unable to halt the progressive scarring that drives the disease, according to a study by researchers at Genethon. Findings suggest that “microdystrophin” gene therapies, similar to several…

Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research and care. Ahead of the event, Bionews, the parent company of this site, sat…

Researchers have identified a molecular pathway that helps regulate how muscle repairs itself — a discovery that may help guide the development of future treatments for conditions such as muscular dystrophy (MD) and severe muscle injuries, a study reports. The pathway depends on a protein called platelet-derived growth factor…

Roche has decided to stop developing satralizumab for bone health in Duchenne muscular dystrophy (DMD), the company announced in a community letter. Patients already enrolled in the  SHIELD DMD Phase 2 trial (NCT06450639) may continue on the study until the six-month bone mineral density collection, expected…

Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their ability to swallow after receiving the gene therapy candidate BB-301 in an ongoing clinical trial. That’s according to an update from therapy developer Benitec Biopharma, which announced new…