Three advocacy organizations are teaming up to run a clinical trial to test if efgartigimod, an approved treatment for certain…
Articles by Marisa Wexler, MS
Avidity Biosciences is planning to double the dosage of AOC 1001 being given to a dozen people with…
An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for…
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…