Children with myotonic dystrophy type 1 (DM1) showed a decline in cognitive function over two years, with frequent impairment…
Articles by Steve Bryson, PhD
Dyne Therapeutics’ FORCE platform delivered its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) 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 (MDA) has announced the schedule for the 2022 MDA Clinical & Scientific Conference, being…
The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical…
A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial…
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan…
Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne…