News

The investigational therapy MA-0211, also called MTB-1, a potential treatment for Duchenne Muscular Dystrophy (DMD), was approved for a Phase I clinical trial, developer Mitobridge announced. The trial will evaluate the therapy’s safety and tolerability and determine its pharmacokinetics and pharmacodynamics profile, or its behavior in the body and mechanism…

The first patient has been dosed in a Phase 2 clinical trial of ACE-083 as a treatment for the muscle impairment in Charcot-Marie-Tooth disease, according to the therapy’s developer, Acceleron Pharma. Acceleron is already evaluating ACE-083 in another Phase 2 trial — this one as a treatment for facioscapulohumeral muscular…

Continuing an 11-year tradition, the Muscular Dystrophy Association (MDA) will once again team up with Casey’s General Stores in Iowa to help send children with muscular dystrophy (MD) and related diseases away for a week of fun and friendship to MDA Summer Camp at no cost to their families. During…

Jiffy Lube and the Muscular Dystrophy Association (MDA) are teaming up again this summer for the 6th annual MUSCLE UP! campaign. Each August for the past five years, Jiffy Lube has campaigned in support of MDA-funded muscular dystrophy and related neuromuscular disease research and programs to help…

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…