Marisa Wexler, MS,  senior science writer—

Avidity Biosciences is planning to double the dosage of AOC 1001 being given to a dozen people with myotonic dystrophy type 1 (DM1) who are receiving the experimental therapy in the MARINA-OLE clinical trial. The U.S. Food and Drug Administration (FDA) had placed a partial clinical hold…

An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). While the FDA is not obligated to abide by the committee’s vote, the agency will consider…

An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting  Friday to discuss SRP-9001 (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD) that’s up for possible approval. The meeting, which will be livestreamed on YouTube, is scheduled from 9 a.m. to…

After two years of treatment with losmapimod, people in a clinical trial with facioscapulohumeral muscular dystrophy (FSHD) showed no upper limb function worsening. Leo Wang, MD, PhD, of the University of Washington, presented the findings at the American Academy of Neurology (AAN) annual meeting in the talk, “…

Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD) one year after being given the gene therapy SRP-9001 (delandistrogene moxeparvovec) continued for a second year, according to new clinical trial data presented at the recent American Academy of…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for Duchenne muscular dystrophy (DMD) that is in early clinical trials. The FDA gives this designation to investigational treatments that have the potential to address unmet clinical care needs for…

A rare neurological problem that does not appear to be directly related to AOC 1001 — the experimental treatment for myotonic dystrophy type 1 (DM1) being given patients in a Phase 1/2 clinical trial — may be the reason for a partial hold placed on the study,…

Treatment with Translarna (ataluren) helps to slow the decline in motor function for people with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, according to new clinical trial data and analyses presented this month at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. The work was funded…

Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s MDA Clinical & Scientific Conference. Three boys with Duchenne muscular dystrophy (DMD) treated with the experimental cell therapy DT-DEC01 in a clinical trial continue to show improvements in motor…

Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in a proof-of-concept clinical trial continue to show improvements in motor function four years after dosing. That’s according to new data discussed this month at the Muscular Dystrophy Association’s MDA Clinical…