Marisa Wexler, MS, senior science writer —

EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation for Duchenne muscular dystrophy (DMD) by the U.S. Food and Drug Administration (FDA). The agency gives this designation to experimental therapies that have the potential to fill unmet medical needs, with the…

Cognitive function in boys with Becker muscular dystrophy (BMD) appear to be stable over time, although these children may struggle with working memory and executive function, a small study reports. Based on its findings, the researchers are calling for boys with BMD to have access to routine cognitive and…

SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping — a trial finding its developers suggest will lead to a positive risk-benefit profile for the next-generation DMD treatment. The full results of the two-part…

SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined…

New trial data announced by Dyne Therapeutics show two experimental treatments for muscular dystrophy from the company’s FORCE platform appear to be working as intended in early clinical studies. Both DYNE-251 for Duchenne muscular dystrophy (DMD), being tested in a trial dubbed DELIVER, and DYNE-101 for…

People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using a wheelchair, a new study reports. The study, “Natural history of Becker muscular dystrophy: a multicenter study of 225 patients,” also provides new insights into the frequency of…

The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD). EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible…

Satellos Bioscience is hoping to bring SAT-3247, its newly nominated lead treatment candidate for Duchenne muscular dystrophy (DMD), into clinical trials next year. The company announced that it’s working to establish a good manufacturing practice (GMP) setup to make the medication, and is in the midst of experiments…

The expression of a protein called ACTC1 in muscle fibers is critical for muscle repair — and vital to the survival of transplanted human muscle stem cells — a new study shows. These findings may have important implications for the development of cell therapies for muscular dystrophy and other…