The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy…
Articles by Marisa Horak, MS
An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the…
The next Clinical & Scientific Conference hosted by the Muscular Dystrophy Association (MDA) will take place March 21-24, 2027,…
Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne…
The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for…
CureDuchenne, a U.S.-based nonprofit that funds research into new treatments for Duchenne muscular dystrophy…
Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed…
The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval…
Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or…
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen…