Marisa Wexler, MS,  senior science writer—

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,”…

GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in…

Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could support the therapy’s accelerated approval by the U.S. Food and Drug Administration (FDA). Regenxbio has also announced…

The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical trial to test ATA-200 — the company’s gene therapy candidate for limb-girdle muscular dystrophy type 2C/R5, or LGMD2C/R5 — in children with this form of LGMD in the U.S.

A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), its developer, Avidity Biosciences, announced. The additional trial cohort, which is expected to be recruited in full early next year,…