Raul Carranza was born in Chula Vista 27 years ago. The recent graduate in political science from the University of California San Diego, and future law student,  is one of the university’s most heralded students. Diagnosed with muscular dystrophy (MD) at the age of two, and by his third birthday…
CureDuchenne, a nonprofit group working to advance research and improve care for people with Duchenne muscular dystrophy (DMD), is partnering with Catabasis Pharmaceuticals to host a webinar discussing promising results from the first part of two-part MoveDMD clinical trial. The webinar is set for Wednesday, June 22, at 1 p.m. ET…
BioBridges, based in Wellesley Hills, Massachusetts, has announced its ongoing support for teen Emily Quitzau and her JettRide fundraiser in July. Emily Quitzau is a 14-year-old triplet with two brothers were both born with severe disabilities. Emily’s brother Calvin was diagnosed with Duchenne muscular dystrophy (DMD) when he was 5…
Researchers at Johns Hopkins Medicine in Baltimore were able to generate muscle cells from skin cells of Duchenne muscular dystrophy (DMD) patients, forming human cell models of different DMD genetic mutations that will allow researchers to further understand the disease and test new therapies. The scientists were also able to correct…
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…
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…
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…
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.
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,…
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