An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for…
Articles by Marisa Wexler, MS
An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting Friday to discuss SRP-9001 (delandistrogene…
After two years of treatment with losmapimod, people in a clinical trial with facioscapulohumeral muscular dystrophy (FSHD) showed…
Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD)…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for…
A rare neurological problem that does not appear to be directly related to AOC 1001 — the experimental treatment for…
Treatment with Translarna (ataluren) helps to slow the decline in motor function for people with Duchenne muscular dystrophy…
Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s…
Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in…
People with Duchenne muscular dystrophy (DMD) who are treated with Exondys 51 (eteplirsen) live significantly longer than would…