Steve Bryson, PhD,  science writer—

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…

The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical trial evaluating DYNE-251, an experimental exon-skipping therapy for Duchenne muscular dystrophy (DMD). Dyne now hopes to have that Phase 1/2 trial underway by June, the company said in a …

A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only been identified as a young man who was not able to walk. In a statement, Pfizer expressed…

RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people…

Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne muscular dystrophy (DMD) who carry nonsense mutations in their DMD gene, according to a study based on real-world data. The investigational therapy also slowed lung function decline by almost two…