News

BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The decision follows deliberations at the May 2016 Committee for Medicinal Products for…

Ryan Benton, the first patient to be approved in the United States for allogenic adult stem cell transplantation as a Duchenne muscular dystrophy (DMD) treatment, turned 30 this year — largely exceeding his original life expectancy. DMD is a genetic disease that affects 1 out of every 3,600 male infants, leading to…

Sarepta Therapeutics of Cambridge, Massachusetts, reports it has been notified by the Food and Drug Administration (FDA) that the agency was unable to complete its review of Sarepta’s New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) drug candidate eteplirsen by the Prescription Drug User Fee Act (PDUFA)…

Nicotinamide riboside (NR), a compound that has been shown effective in boosting metabolism, can also induce muscular regeneration and enhance life span in mice, according to the study “NAD+ repletion improves mitochondrial and stem cell function and enhances life span in mice,” published in…

Genea Biocells has developed a human stem cell-based cellular model for a type of muscular dystrophy, known as facioscapulohumeral muscular dystrophy (FSHD), that may be a suitable tool for high-throughput screening and the development of therapeutic agents for the disease. The model is described in the study “A…

Switzerland-based Santhera Pharmaceuticals‘ drug candidate omigapil has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophies (CMD). Omigapil is a prenyl-analog with anti-apoptotic properties. Nonclinical studies in disease-relevant models show the drug inhibits cell death and reduces body weight loss and skeletal…

Scientists for a first time have captured the entire process of muscle regeneration — from injury to fiber replacement — in a living animal, validating the role of specific stem cells in regeneration. Their work could lead to new ways of triggering this process, so as to better treat people with muscle wasting…

Researchers at the UCLA Center for Duchenne Muscular Dystrophy and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have been working on a new approach that can potentially be used to treat Duchenne muscular dystrophy (DMD). “This method is likely 10 years away…

More than 50 international experts came together last week in a workshop organized by the Parent Project Muscular Dystrophy (PPMD), to discuss bone health and osteoporosis in Duchenne muscular dystrophy (DMD). The two-day workshop on May 12-13 brought together scientists, clinicians, and industry representatives from around the world. Different aspects…

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization in the fight to end Duchenne muscular dystrophy (DMD), has confirmed Children’s Mercy Hospital in Kansas City, Mo., as a Certified Duchenne Care Center. “I am very proud of our multidisciplinary team,” said Dr. Ann Modrcin, director of the…