News

The U.S. Food and Drug Administration (FDA) has granted Capricor Therapeutics rare pediatric disease designation for CAP-1002, its candidate therapy to treat  Duchenne muscular dystrophy (DMD). This latest approval, along with the FDA’s recent approval of orphan drug status to CAP-1002, “underscores the urgent need for treatment options for this devastating…

With nearly 7,000 rare diseases, including Duchenne muscular dystrophy, affecting an estimated 30 million Americans, it’s hard for Paul Melmeyer to keep track of them all. As director of federal policy at the National Organization for Rare Disorders (NORD), Melmeyer’s job is to represent the rare disease patient in…

Parent Project Muscular Dystrophy (PPMD), a nonprofit working to end Duchenne muscular dystrophy (DMD), has recognized Gillette Children’s Specialty Healthcare Neuromuscular Clinic as a Certified Duchenne Care Center. The Certified Duchenne Care Center Program was launched by PPMD in March 2014 as part of its Transforming Duchenne Care Initiative.

Sarepta Therapeutics has partnered with Idis Managed Access, a division of Clinigen Group, to develop a managed access program (MAP) for its lead therapy Exondys 51 (eteplirsen) for the treatment of Duchenne muscular dystrophy (DMD). The MAP was planned to provide early, expanded access to Sarepta’s Exondys 51…

The latest results of the Phase 3 clinical trial for PTC Therapeutics’ candidate Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) showed that it can benefit patients who are in a transition stage of the disease and are able to walk between 300 and…

The Muscular Dystrophy Association (MDA) has awarded Dr. Johanna Hamel, a neurologist at New York’s University of Rochester, a $130,000 fellowship to help her research the underlying mechanisms that cause myotonic dystrophy — the most common form of adult-onset muscular dystrophy. The American Academy of Neurology (AAN) and the American Brain Foundation…

Everest to End Duchenne, an organization raising funds to support research to end Duchenne muscular dystrophy, is looking for hikers, outdoor enthusiasts and climbers to take part in its second trek to Mount Everest Base Camp on Sept. 25. The treks represent the challenge that boys with Duchenne…

The U.S. Food and Drug Administation has granted orphan designation to Mallinckrodt Pharmaceuticals‘ drug candidate MNK-1411 for treatment of Duchenne Muscular Dystrophy (DMD). MNK-1411 is an injection composed of a formulation of tetracosactide, which is a synthetic melanocortin receptor agonist. Melanocortin receptor agonists are hormones that activate melanocortin receptors and…

The molecular process that leads to congenital myotonic dystrophy (CMD) in infants and children was identified by researchers working with tissue samples and mice. Their study, titled “Disrupted prenatal RNA processing and myogenesis in congenital myotonic dystrophy,” was published in the journal Genes and Development. Genes code for various proteins that…

A comprehensive 12-year genetic and clinical analysis of unrelated patients with congenital muscular dystrophies (CMDs) in the U.K. has confirmed MDC1A as the the most common CMD subtype, and identified 160 new mutations. The study, “Congenital muscular dystrophies in the UK population: Clinical and molecular spectrum of a large…