News

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…

Parent Project Muscular Dystrophy (PPMD) recently recognized Children’s Hospital Los Angeles as a Certified Duchenne Care Center (CDC). The hospital is one of only 10 facilities in the U.S. to hold this distinction and the only one in Southern California since the PPMD began qualifying healthcare centers in 2014.

Promising new biomarkers to detect structural changes in the hearts of people with Duchenne and Becker muscular dystrophy have been identified, according to a study published in the Journal of Cardiovascular Magnetic Resonance. Biomarkers, like miRNAs, that circulate in the blood and are easily detectable can help diagnose and monitor chronic heart-muscle disease, or…