José Lopes, PhD, managing science editor —

The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) treatment, its developer, Wave Life Sciences, announced. WVE-210201 is an investigational compound of a type of therapy called…

People with Duchenne muscular dystrophy (DMD) in the U.K. will continue to have access to Raxone (idebenone) to treat their disease, after the Medicines and Healthcare products Regulatory Agency (MHRA) renewed its Early Access to Medicines Scheme (EAMS) with the therapy’s developer, Santhera Pharmaceuticals. Raxone has been available in the…

A quantitative method that can detect changes in ankle muscle strength in patients with myotonic dystrophy type 1 (MD1) was found to be highly accurate, and may work as an accurate and consistent way of assessing patients in clinical trials. The study, “Ankle Strength Impairments in Myotonic…

Researchers identified proteins that regulate DUX4, the key gene in facioscapulohumeral muscular dystrophy (FSHD). The results offer a potential therapeutic pathway, they believe. The study, “NuRD and CAF-1-mediated silencing of the D4Z4 array is modulated by DUX4-induced MBD3L proteins,” appeared in the journal eLife. While healthy…

Researchers have generated skeletal muscle from human stem cells in the laboratory, an achievement that may advance development of treatments and tissue replacement for patients with muscular dystrophy (MD). Those findings are reported in the study, “Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage…

A genetic compensation mechanism discovered in zebra fish could have implications in the treatment of muscle diseases, a recent study suggests. The study, “Genetic compensation triggered by actin mutation prevents the muscle damage caused by loss of actin protein,” appeared in the journal PLOS Genetics. Many…

The use of tamoxifen and Evista (raloxifene) improved cardiac, respiratory, and skeletal muscle functions, and increased bone density in a mouse model of muscular dystrophy (MD), research from the Carolinas Medical Center suggests. The study, “Long-Term Treatment of Tamoxifen and Raloxifene Alleviates Dystrophic Phenotype and Enhances Muscle Functions of FKRP Dystroglycanopathy,”…

AMO Pharma’s myotonic dystrophy treatment improved patients’ cognition, fatigue and  ability to perform daily tasks, a Phase 2 clinical trial showed. The therapy, AMO-02 (tideglusib), also improved patients’ autism symptoms. In addition, it was safe and patients tolerated it well. Dr. Joseph Horrigan, the company’s chief medical officer, presented the findings at…

Sarepta Therapeutics will seek accelerated U.S. approval of a treatment for a subset of Duchenne muscular dystrophy (DMD) patients. Golodirsen (SRP-4053) is for patients whose disease stems from a deleted part of the DMD gene known as exon 53. Sarepta met with U.S. Food and Drug Administration officials in…

Research supporting prenatal diagnosis of Duchenne muscular dystrophy (DMD) has earned Vietnamese scientist Tran Van Khanh a Kovalevskaya Award. Khanh has focused her work on studying gene therapy and prenatal diagnosis of genetic disorders. Germany’s Alexander von Humboldt Foundation makes the annual Kovalevskaya Awards to women scientists in…