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…
News
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…
September is Muscular Dystrophy Awareness Month, and advocates are launching campaigns to educate people about the different types of rare genetic muscle disorders that comprise muscular dystrophy (MD) — and to fundraise to fuel research. Still, the focus is on spotlighting those living with MD, Kelly Berger, community engagement…
For the first time, a person with Duchenne muscular dystrophy (DMD) has been given an experimental gene-editing therapy, called PBGENE-DMD, that’s designed to treat the neurodegenerative disease by altering the patient’s own genetic code. Precision Biosciences, the U.S. company developing PBGENE-DMD, announced that dosing had begun in the…
The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from…
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