News

Never before has the therapeutic pipeline of potential treatments for Duchenne muscular dystrophy appeared so promising. Yet that also means more questions than ever. Searching for answers, about 620 people — 70 percent of them parents — converged on Phoenix, Arizona, in late June for the 2018 Annual Conference organized…

Discussions of the work being done on four different approaches to treating Duchenne muscular dystrophy (DMD) — repairing or replacing dystrophin, managing inflammation, improving muscle mechanics, and editing dysfunctional genes using CRISPR/CAS9 — were a highlight of sessions at the recent 2018 Parent Project Muscular Dystrophy (PPMD) conference in Scottsdale, Arizona.

Cognitive behavioral therapy improved physical activity and social participation in patients with myotonic dystrophy type 1, indicating its potential as a treatment method for these patients, according to results from a clinical trial. Findings were reported in the study, “Cognitive behavioural therapy with optional graded exercise therapy in…

Summit Therapeutics is stopping the clinical development of ezutromid, which it was investigating as a Duchenne muscular dystrophy (DMD) treatment, after a Phase 2 clinical trial testing the therapy failed to reach both its primary and secondary objectives. “These data come as a great disappointment to us and to…

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…

Microdystrophin gene therapy robustly induces the production of a shorter, but functional, version of the dystrophin protein and reduces muscle damage in Duchenne muscular dystrophy (DMD) patients, according to preliminary results of a Phase 1/2 clinical trial. DMD is a severe type of muscular dystrophy caused by mutations in the…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, a dystrophin gene therapy aiming to treat Duchenne muscular dystrophy (DMD). FDA, in its letter, said that Solid Biosciences, the therapy’s developer, had addressed all questions of concern that led to…

The Centers for Disease Control and Prevention (CDC) released new, updated guidelines for the management of Duchenne muscular dystrophy (DMD) patients. Researchers highlighted the role of rehabilitation techniques and the importance of physiatrists to improve function and quality of life. The study,…

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…