Betty Vertin had never heard of Duchenne muscular dystrophy before July 8, 2010 — the day a neurologist in Omaha, Nebraska, diagnosed her son Max with the rare disease. “Will you spell that for me?” she remembers asking the doctor through her tears. Vertin was 10 weeks pregnant when she…
With so much focus on the very real dangers of muscular, pulmonary and cardiac decline, it’s easy for moms and dads of Duchenne kids to forget about less urgent issues like dental health and digestion. But they shouldn’t, said experts speaking at Parent Project Muscular Dystrophy’s recent 2018…
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…
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…
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