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…
News
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…
An immune signaling pathway involving type I interferon (IFN-I) may be a potential therapeutic target for muscle damage in people with myotonic dystrophy type 1 (DM1), a new study suggests. An examination of immature muscle cells from children with DM1 found that increased IFN-I signaling was associated with stress…
Mindy Henderson has spent much of her life challenging the limits others have placed on her. When she was diagnosed with spinal muscular atrophy (SMA) — a genetic condition that causes muscles to weaken over time — as an infant, doctors told her parents that she might not live long…
Twenty college students with Duchenne muscular dystrophy (DMD) will receive awards of up to $5,000 through Sarepta Therapeutics‘ Route 79 program. “As we mark the ninth year of this program, we continue to be inspired by the commitment these students show to their education and their futures,” Diane…
A Phase 3 clinical trial testing delpacibart etedesiran (del-desiran), an experimental treatment for myotonic dystrophy type 1 (DM1), failed to hit its main goal. The Phase 3 HARBOR trial (NCT06411288) enrolled nearly 160 people with DM1, ages 16 to 65. Participants received seven infusions of either del-desiran or…
Scientists now have evidence to explain why treatment with the corticosteroid prednisone — but not the newer steroid Agamree (vamorolone) — drives bone loss in young boys with Duchenne muscular dystrophy (DMD) and may slow their growth. Using data from a clinical trial, the researchers found that…
The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body. The experimental…
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