A large clinical trial evaluating the treatment candidate sevasemten for people with Becker muscular dystrophy (BMD) has failed to meet its main goal, with “no statistically significant benefit” seen on a measure of physical function. That’s according to top-line data, or the first study results, from GRAND…

Treatment with the experimental gene therapy BB-301 was found to improve swallowing ability for every person with a rare type of muscular dystrophy (MD) that mainly affects the throat and eyes who are taking part in a first-in-human clinical trial. That’s according to new interim data announced by Benitec…

The Muscular Dystrophy Association (MDA) is hailing a recent U.S. federal court ruling that blocks, at least for now, the implementation of new restrictions that would have made it harder for many people with disabling conditions such as muscular dystrophy to qualify for Medicaid, the government-run program that…

Two interconnected themes — community and hope — dominated the conversations at a recent gathering of people living with neuromuscular diseases. Lily Sander, MDA National Ambassador, speaking at MDA Engage: Dallas. (Photo by Tara Haelle) Lily Sander, national ambassador for the Muscular Dystrophy Association (MDA), hit on those…

The first step to figuring out the right equipment for someone with a neuromuscular disease like muscular dystrophy is to understand that person’s goals, environment, and changing needs, a pair of experts said. “There’s a lot of equipment out there,” said Katie Metheny, an occupational therapist based in the…

A new gene therapy approach safely restored the production of emerin, the protein missing in people with X-linked Emery-Dreifuss muscular dystrophy (EDMD), in both a mouse model and human muscle cells, according to a study. Although the preclinical study could not assess the gene therapy’s impact on muscle function,…

Dyne Therapeutics‘ experimental therapy zeleciment basivarsen (z-basivarsen) may help improve multiple measures of physical function in people with myotonic dystrophy type 1 (DM1), new clinical trial data suggest. “What’s encouraging is that we observed improvements across multiple measures, including strength and functional assessments,” Doug Kerr, MD, PhD, chief…

Mental health problems such as anxiety and depression are common for people with muscular dystrophy, affecting most individuals in the community, according to a new survey. The survey was carried out by Bionews, the parent company of Muscular Dystrophy News. Survey results indicate that loneliness, regret, and…

Lily Sander will never forget what it was like to meet other people with neuromuscular diseases the first time she attended Muscular Dystrophy Association (MDA) Summer Camp. Living with Charcot-Marie-Tooth disease, Sander had met only one or two other people with neuromuscular diseases in her city. She was…

The gene therapy Elevidys (delandistrogene moxeparvovec-rokl), approved in the U.S. to treat people ages 4 and older with Duchenne muscular dystrophy (DMD), can be safely administered to boys with DMD as young as 2 years old, new data show. A new analysis, which covered nine boys with DMD…