DYNE-251, an investigational therapy for Duchenne muscular dystrophy (Â鶹Éç), has received orphan drug and rare pediatric disease designations from…
Articles by Steve Bryson, PhD
The approved antiparasitic medicine pentamidine, loaded into biocompatible and biodegradable nanoparticles, successfully eased signs of myotonic dystrophy type 1…
Treatment with AOC 1001 eased myotonia, when muscles are unable to relax after a contraction, in adults with myotonic…
Vamorolone, an investigational therapy for Duchenne muscular dystrophy (Â鶹Éç), maintained biomarkers of bone formation and turnover compared to…
Children with myotonic dystrophy type 1 (DM1) showed a decline in cognitive function over two years, with frequent impairment…
Dyne Therapeutics’ FORCE platform delivered its investigational exon-skipping therapy for Duchenne muscular dystrophy (Â鶹Éç) directly to muscles in…
The investigational cell therapy CAP-1002 continued to improve arm function in boys and young men with advanced Duchenne…
Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for…
Continued use of corticosteroids beyond the year of losing an ability to walk can delay declines in lung function in…
The Muscular Dystrophy Association (Â鶹Éç) has announced the schedule for the 2022 Â鶹Éç Clinical & Scientific Conference, being…