If it weren’t for a serious auto accident during his senior year of high school, Christopher Anselmo might have had to wait many more years before discovering he had limb-girdle muscular dystrophy (LGMD). This rare condition — a general term for more than 30 muscle-wasting conditions — affects anywhere from…
Parent Project Muscular Dystrophy (PPMD) has published a report outlining key results of its decade-old study of some 4,000 registered Duchenne and Becker muscular dystrophy patients. Representing more than 100 countries, the nonprofit organization’s Ten Year Registry Report is a compilation of data provided by…
To improve patient engagement and care management tools for the Duchenne Registry, Parent Project Muscular Dystrophy (PPMD) has teamed up with THREAD, a company specializing in remote and virtual patient research. Enhancements to the registry — available on both IOS and Android devices — will be mainly…
The U.S. Food and Drug Administration (FDA) selected  Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 (suvodirsen) in Duchenne muscular dystrophy for its pilot program for complex innovative trial designs. This initiative aims to modernize the design of clinical trials and advance drug development. Wave will…
Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Aldevron, which produces biological products for the biotech industry, will be responsible for supplying…
Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) clinical trials were turning studies away — they didn’t have the resources to test all of the new and potential advancements. But starting last year, the Great Ormond Street Hospital in…
Kanneboyina Nagaraju, PhD, and his research team at Binghamton University, State University of New York will get a $329,000 Parent Project Muscular Dystrophy (PPMD) grant to study responses to differing treatments for Duchenne muscular dystrophy. The researchers will focus on the role of the immune system…
A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy (DMD) at multiple sites in the U.S. and will soon open sites worldwide, according to Catabasis Pharmaceuticals. Nine sites across the U.S. are now enrolling participants for this one-year,…
Capricor Therapeutics put a voluntary hold on a Phase 2 clinical trial of its Duchenne muscular dystrophy (DMD) investigative cell therapy CAP-1002 after a patient experienced a severe allergic reaction during blind testing. According to a recent U.S. Securities and Exchange Commission report, Capricor halted its HOPE-2 (NCT03406780) study,…
The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for limb-girdle muscular dystrophy type 2D (LGMD 2D), the company announced. The FDA grants this designation to treatments with the potential to significantly improve the life of…
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