Marisa Wexler, MS,  senior science writer—

The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend Translarna (ataluren) be covered to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations. According to NICE, although there’s evidence Translarna can benefit people with DMD, it’s not worth…

Sarepta Therapeutics has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to grant accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) as a treatment for Duchenne muscular dystrophy (DMD) in patients who are able to walk. “Every hour of every…

Four months of treatment with the experimental oral therapy EDG-5506 reduced markers of muscle damage and improved measures of physical function among men with Becker muscular dystrophy (BMD) in the Phase 1 ARCH clinical trial, new data show. Edgewise Therapeutics, the company developing EDG-5506, recently launched a Phase…

For the fourth year, a family in Montana is hosting “Calves to Cure,” a cattle auction that aims to raise funds to support developing new treatments, or even a cure, for Duchenne muscular dystrophy. The event will take place Sept. 15 in Billings, Montana. All proceeds from the…

Under an agreement with the Broad Institute, Sarepta Therapeutics has acquired an exclusive license to the gene therapy vector MyoAAV in potentially treating Duchenne muscular dystrophy (DMD) and several other neuromuscular and cardiac indications. MyoAAV is a new vector for delivering genetic therapies to cells, with evidence that…

Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant accelerated approval to the company’s experimental gene therapy SRP-9001 for people with Duchenne muscular dystrophy (DMD) who are able to walk. “We are delighted to confirm that based on the feedback we received…

The first participant has been dosed in the Phase 3 REACH clinical trial testing the safety and effectiveness of Fulcrum Therapeutics‘ experimental oral therapy losmapimod in people with facioscapulohumeral muscular dystrophy (FSHD). If results from the trial are positive, they’re expected to support applications for lospmapimod’s approval…

Pliant Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) for permission to begin clinical testing of PLN-101325, its experimental therapy for muscular dystrophy, by the end of this year. In preclinical studies involving cell and animal models — the results of which were presented…

The experimental oral therapy EDG-5506 has been generally well-tolerated and lowered markers of muscle damage among men with Becker muscular dystrophy (BMD) in the open-label ARCH clinical trial, early data show. Findings supported the shift to a higher daily treatment dose in study patients. “These interim 2-month ARCH data…

The Muscular Dystrophy Association (MDA) is hosting a one-day games streaming fundraiser, dubbed “MDA Rivals,” this Saturday, June 18. The event, streaming on MDA’s Twitch channel, will feature stars from MDA Let’s Play — a platform that aims to raise funds for MDA and awareness about muscular…