Marisa Wexler, MS,  senior science writer—

Santhera and ReveraGen have completed their rolling application asking the U.S. Food and Drug Administration (FDA) to approve the dissociative corticosteroid vamorolone to treat Duchenne muscular dystrophy (DMD). Rolling applications allow companies to submit individual sections of the application as soon as they are completed, rather than waiting…

Treatment with Viltepso (viltolarsen) helps preserve motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to four-year data from an open-label extension study. “These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne…

The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend Translarna (ataluren) be covered to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations.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…