Marisa Wexler, MS, senior science writer —

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…

After 18 months, treatment with the experimental cell therapy CAP-1002 continues to show beneficial effects on upper limb function in people with Duchenne muscular dystrophy (DMD), according to new data from the open-label extension phase of the HOPE-2 study. The new trial data also suggests a consistent safety…

Wave Life Sciences‘ experimental exon-skipping therapy WVE-N531 appears to be working as expected, according to early data from a small clinical trial of the therapy in a first group of three boys with Duchenne muscular dystrophy (DMD). “These data provide early evidence that WVE-N531 is … leading to…

The U.S. Food and Drug Administration (FDA) has given priority review Sarepta Therapeutics‘ application seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec), the company’s experimental gene therapy for Duchenne muscular dystrophy (DMD) in patients who can walk. An FDA decision is expected on or before May 29, 2023. “We are delighted…

PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed promising effects in preclinical studies, the therapy’s developer PepGen announced. The company also announced promising results from cell experiments for two other investigational exon-skipping therapies, PGN-EDO45 and PGN-EDO44.