Marisa Wexler, MS,  senior science writer—

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…

A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), now will be reviewed by the European…

People with myotonic dystrophy type 1 (DM1) reported gains in measures of muscle strength after being treated with AOC 1001 in a clinical trial. That’s according to new data announced by Avidity Biosciences, the developer of AOC 1001. The findings “reinforce our belief in the potential of AOC 1001…

The gene therapy RGX-202 at lower dose was well tolerated by three boys with Duchenne muscular dystrophy (DMD) in an early clinical trial, and biomarker data suggest the treatment is working as intended. Regenxbio, the company developing RGX-202, expects to administer a higher dose of…