The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated…
Articles by José Lopes, PhD
People with Duchenne muscular dystrophy (DMD) in the U.K. will continue to have access to Raxone (idebenone) to treat their…
A quantitative method that can detect changes in ankle muscle strength in patients with myotonic dystrophy type 1 (MD1) was…
Researchers identified proteins that regulate DUX4, the key gene in facioscapulohumeral muscular dystrophy (FSHD). The results offer a…
Researchers have generated skeletal muscle from human stem cells in the laboratory, an achievement that may advance development of treatments…
A genetic compensation mechanism discovered in zebra fish could have implications in the treatment of muscle diseases, a recent…
The use of tamoxifen and Evista (raloxifene) improved cardiac, respiratory, and skeletal muscle functions, and increased bone density in a…
AMO Pharma’s myotonic dystrophy treatment improved patients’ cognition, fatigue and ability to perform daily tasks, a Phase 2 clinical trial showed.
Sarepta Therapeutics will seek accelerated U.S. approval of a treatment for a subset of Duchenne muscular dystrophy (DMD) patients.
Research supporting prenatal diagnosis of Duchenne muscular dystrophy (DMD) has earned Vietnamese scientist Tran Van Khanh a Kovalevskaya Award.