News

Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene to muscles as a way to correct the faulty gene that characterizes Duchenne muscular dystrophy (DMD), a mouse study shows. Their study, “A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic…

The U.S. Food and Drug Administration has approved Capricor Therapeutics’ plan for further development of its CAP-1002 stem cell therapy for Duchenne muscular dystrophy, paving the way for another Phase 2 clinical trial of the treatment this year. The thumbs-up came when Capricor received official minutes from a…

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows. “This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to…

Long after it arrived elsewhere, Emflaza (deflazacort) became the first corticosteroid that the U.S. Food and Drug Administration approved to treat all forms of Duchenne muscular dystrophy — in February 2017. But its U.S. introduction was far from smooth. In fact, it took three more months and a change of…

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…