News

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…

MDA Southern Wisconsin, together with presenting sponsor Harley-Davidson Motor Company, will host the 23rd Annual Black-N-Blue Ball charitable gala in Milwaukee on May 20, aiming to raise $1 million for adults and children with muscular dystrophy. Jann Carl of “Entertainment Tonight” will serve as master of ceremonies. At the event,…

Wave Life Sciences, Ltd., is planning to begin clinical testing of its next-generation nucleic acid therapy for patients with Duchenne muscular dystrophy (DMD), which the company says has shown promise in pre-clinical trials. Its initial DMD candidate skips exon 51 in the dystrophin gene. Data from early studies showed a roughly…

Researchers have discovered the molecular mechanisms responsible for the heart dysfunctions that mark myotonic dystrophy, and reported they appear to be linked to a defective processing of the cardiac sodium channel (SCN5A) RNA. Their study, published in Nature Communications, is titled “Splicing misregulation of SCN5A contributes to cardiac-conduction delay and…