DYNE-251, an investigational therapy for Duchenne muscular dystrophy (DMD), has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA). Orphan drug status is meant to support the development of therapies for rare conditions affecting less than 200,000 people in the U.S. It…
The approved antiparasitic medicine pentamidine, loaded into biocompatible and biodegradable nanoparticles, successfully eased signs of myotonic dystrophy type 1 (DM1) in cell-based models, a study shows. The researchers noted that these findings show the potential of nanoparticles to deliver therapeutic agents to treat muscle cells in people with DM1…
Treatment with AOC 1001 eased myotonia, when muscles are unable to relax after a contraction, in adults with myotonic dystrophy type 1 (DM1), according to early data from the Phase 1/2 MARINA clinical trial. MARINA data also showed — in all treated participants — AOC 1001 was successfully delivered…
Vamorolone, an investigational therapy for Duchenne muscular dystrophy (DMD), maintained biomarkers of bone formation and turnover compared to prednisone, according to the now published details of the VISION-DMD study. Standard anti-inflammatory corticosteroids, such as prednisone, have been shown to stunt growth with long-term use. Vamorolone is a…
Children with myotonic dystrophy type 1 (DM1) showed a decline in cognitive function over two years, with frequent impairment in visuospatial skills and attention, a small study suggested. The data indicate cognitive, neuropsychological, emotional, and behavioral assessments should be administered to children with DM1 periodically during development, the researchers…
Dyne Therapeutics’ FORCE platform delivered its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) directly to muscles in a mouse model of the disease, according to the results of a preclinical study. The potential DMD treatment improved dystrophin protein production and enhanced physical activity in treated mice. The…
The investigational cell therapy CAP-1002 continued to improve arm function in boys and young men with advanced Duchenne muscular dystrophy (DMD), according to the HOPE-2 open-label extension study. These additional upper limb improvements came after HOPE-2 participants were off CAP-1002 for a mean of about one year before…
Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for Duchenne muscular dystrophy (DMD). The move was based on recent preclinical data, the company announced in a press release. AT702, AT751, and AT753 are exon skipping medicines designed…
Continued use of corticosteroids beyond the year of losing an ability to walk can delay declines in lung function in boys with Duchenne muscular dystrophy (DMD), an analysis of MD STARnet data suggested. In comparison, patients who never used corticosteroids or who discontinued treatment within one year of losing ambulation…
The Muscular Dystrophy Association (MDA) has announced the schedule for the 2022 MDA Clinical & Scientific Conference, being held March 13-16, both via live stream and in person at the Gaylord Opryland Resort and Convention Center in Nashville, Tennessee. The event, to be covered by Muscular Dystrophy…
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