Acceleron’s ACE-083, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA), the company announced. FSDH affects about 20,000 people in the U.S. It is a rare genetic muscle disorder for which there…
Five years after the death of a Utah boy named Mitchell Jones, the national foundation created in his name helps families struggling with the ravages of Duchenne muscular dystrophy. Mitchell, who was diagnosed with Duchenne at the age of 3, died of heart failure on March 2, 2013, just…
A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will begin enrolling boys, ages 4 to 7, in the coming months, Catabasis Pharmaceuticals announced. Top-line results from this trial, to be called POLARIS, are expected in April 2020, and this…
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…
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