Marisa Wexler, MS,  senior science writer—

Five people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakness affecting the face and throat — have thus far been treated with the gene therapy candidate BB-301 in an ongoing clinical trial, treatment developer Benitec Biopharma said in a…

A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of healthy volunteer enrollment in our Phase 1 trial marks a significant milestone in our mission to develop transformative…

A fundraiser through the online role-playing game World of Warcraft has raised more than $2 million to support CureDuchenne, an organization dedicated to combating Duchenne muscular dystrophy (DMD). The fundraiser, which launched last year in partnership with Warcraft’s maker Blizzard Entertainment, is the largest contribution from a…

Treatment with oral therapy ifetroban led to improvements in measures of heart function in people with Duchenne muscular dystrophy (DMD), according to top-line results from a clinical trial. The “impressive results” are a “pivotal moment” for developer Cumberland Pharmaceuticals, “and, more importantly, for the DMD community,” A.J. Kazimi,…

The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood — that’s now being tested in a clinical trial involving DM1 patients. This status is awarded by the…

Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD). “The submission of the BLA marks a pivotal step for Capricor and those impacted by…

The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases. Altogether, the funding totals more than $5 million across 21 grants, according to a press release…

The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD) treated in a Phase 2 trial, according to top-line results announced by the therapy’s developer, Edgewise Therapeutics. “This landmark study presents compelling biomarker data and promising signals that…

The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who are working to develop new treatments for facioscapulohumeral muscular dystrophy (FSHD). “This competition will bring together the brightest minds in medicine, technology, and science, all working toward a cure for this devastating disease,”…