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…
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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…
Boys and young men with Duchenne muscular dystrophy (DMD) who switched from a placebo to the experimental cell therapy deramiocel as part of a clinical trial showed smaller declines in measures of upper limb function after making the change, new data show. Data from the study, which included…
The U.S. Food and Drug Administration (FDA) has given the go-ahead for a mid-stage clinical trial testing the oral therapy forazapadin — designed to protect muscles and potentially enhance their regeneration — in adults with facioscapulohumeral muscular dystrophy (FSHD), marked by muscle weakness that particularly affects the face,…
For many people living with rare neuromuscular diseases, the most meaningful part of a recent Chicago gathering was not just the medical updates — it was finding a community that understood their daily struggles. The realization that they were not alone set the tone for MDA Engage: Chicago, a…
New treatments for muscular dystrophy (MD) are moving forward, but no single therapy will work for every MD type, and some promising therapies have fallen short in trials, two neuromuscular specialists told patients and families. “Muscular dystrophies are not just one condition,” said Ryan Jacobson, MD, a neuromuscular…
People living with neuromuscular diseases and their families should help shape new treatments from the very start, rather than weighing in only after key decisions are made, according to experts and community members who spoke on a Sept. 26 panel at the MDA Engage: Chicago symposium. The panel, “…
A protein-rich diet was associated with less body fat and better quality of life among adults with Becker muscular dystrophy (BMD), a small study in the U.K. found. While the study didn’t show that eating more protein would reduce body fat or improve quality of life, the findings “support…
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