Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD)…
Articles by Marisa Wexler, MS
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…
Researchers are planning to enroll a new group of patients in a natural history study to collect data about how…
A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular…
Protecting structures on the end of chromosomes, called telomeres, by increasing levels of the protein TRF2, was found to improve…