News

Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial results also demonstrate that measuring the air flow speed in and out of the lungs, or peak expiratory flow…

Benitec Biopharma recently granted Axovant Sciences worldwide rights to its investigational AXO-AAV-OPMD gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) under a global licensing agreement. AXO-AAV-OPMD is currently in preclinical development, and Axovant plans to launch a placebo-controlled trial testing the therapy candidate for OPMD…

Coping with Duchenne muscular dystrophy is difficult enough in the United States. But in neighboring Mexico, people with Duchenne often face social discrimination, lack of access to the newest therapies, a financially strapped healthcare system, and general ignorance. “Very few Mexicans know anything about this disease,” said Graciela Méndez Covarrubias,…

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…