Marisa Wexler, MS,  senior science writer—

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…

While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results nonetheless showed that, compared with a placebo, the approved gene therapy led to notable improvements across numerous measures of physical function in boys with Duchenne muscular dystrophy (DMD). Top-line findings…

DYNE-251, an experimental treatment for Duchenne muscular dystrophy (DMD), is being well tolerated by patients in an ongoing clinical trial, according to the therapy’s developer, Dyne Therapeutics. Dyne also reported positive safety data from an ongoing trial of DYNE-101, the company’s investigational therapy for myotonic dystrophy type…

A gene editing approach can restore dystrophin production in cell and animal models of Duchenne muscular dystrophy (DMD), according to its researchers. The study, “Targeting Duchenne Muscular Dystrophy by Skipping DMD Exon 45 with Base Editors,” was published in Molecular Therapy Nucleic Acids. DMD is caused by…