Emma Yasinski,  —

DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK

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…

PPMD Webinar Takes In-depth Look at MD Cardiac Disease and Recent Research Efforts

Magnetic Resonance Imaging (MRI) can catch signs of damage to the heart in muscular dystrophy (MD) patients earlier than traditionally-used echocardiograms, according to a webinar providing updates on the Parent Project for Muscular Dystrophy (PPMD)’s cardiac initiative. The presentation took place Dec 12, which is almost halfway through…

Phase 3 Trial of Edasalonexent in Boys with DMD and Any Mutation Enrolling in US, Catabasis Reports

A pivotal Phase 3 trial of edasalonexent, a potential oral disease-modifying treatment for Duchenne muscular dystrophy (DMD) regardless of mutation type, is enrolling boys ages 4 to 7 in the U.S. and will soon begin with those in Australia, Canada, Europe, and Israel. “We named the trial ‘Polaris’ because it…

4 Approaches to Treating Duchenne Muscular Dystrophy Highlighted at PPMD Conference

Discussions of the work being done on four different approaches to treating Duchenne muscular dystrophy (DMD) — repairing or replacing dystrophin, managing inflammation, improving muscle mechanics, and editing dysfunctional genes using CRISPR/CAS9 — were a highlight of sessions at the recent 2018 Parent Project Muscular Dystrophy (PPMD) conference in Scottsdale, Arizona.