Marisa Wexler, MS, senior science writer —

Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as it should, making it unlikely to raise a person’s COVID-19 risk, Catabasis Pharmaceuticals said. COVID-19 is believed to pose the greatest threat to older people and those…

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a treatment for facioscapulohumeral muscular dystrophy (FSHD). The data, “Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability and Target Engagement,” were presented by Michelle…

Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the COVID-19 pandemic. Also, the company continues to work with regulatory authorities to guarantee the continuation…

Machine learning could be used to help diagnose specific forms of muscular dystrophy (MD) using magnetic resonance imaging (MRI) scans, a study suggests. The study, “Accuracy of a machine learning muscle MRI-based tool for the diagnosis of muscular dystrophies,” was published in the journal…

Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD). Capricor’s lead candidate for DMD is CAP-1002, which is composed of cardiosphere-derived cells (CDCs). These are cardiac progenitor cells, meaning they are able to…

A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…

Supplementing the diet with the amino acid L-arginine may improve gut function in people with Duchene Muscular Dystrophy (DMD), a study in mice suggests. The study, “Spatiotemporal Mapping Reveals Regional Gastrointestinal Dysfunction in Mdx Dystrophic Mice Ameliorated by Oral L-arginine Supplementation,” was published in the …

Addressing changes in cellular metabolism and oxidative damage may lead to muscle regeneration in Duchenne muscular dystrophy and Becker muscular dystrophy, a study has found. The study, “Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle…

Translarna (ataluren) significantly improves physical fitness in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), a study examining multiple endpoints across two trials shows. The study used a statistical strategy for analyzing multiple trial endpoints together, a tactic that could improve the interpretation…

A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize how the brain is affected in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), and to find potential ways to treat neurological complications in people with those disorders. The European Union awarded…