Marisa Wexler, MS,  senior science writer—

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…

People with Duchenne muscular dystrophy (DMD) who are treated with Exondys 51 (eteplirsen) live significantly longer than would be expected without the treatment, by a median of more than five years, according to a new analysis. The longest survival times were generally seen among patients who started on…

Researchers are planning to enroll a new group of patients in a natural history study to collect data about how Duchenne muscular dystrophy (DMD) progresses and affects a person’s life. Natural history studies don’t test an experimental therapy, but instead work to collect detailed information on how a disease…

A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular dystrophy (LGMD), a new study reports. “We show an effective and precise repair to correct the most frequent [LGMD type 2A] mutation … Our approach is a promising source for…

Protecting structures on the end of chromosomes, called telomeres, by increasing levels of the protein TRF2, was found to improve heart cell survival and function in a model of Duchenne muscular dystrophy (DMD), a new study reports. “Our findings suggest that preserving telomere lengths plays a critical role in…

A new dominant-negative tumor necrosis factor (DN-TNF) treatment reduced muscle damage and promoted muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), according to INmune Bio. The research is part of a collaboration between INmune and Armando Villalta, PhD, a professor at the University California, Irvine…