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January 30, 2019February 6, 2019

ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies

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The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD). The review, “Deflazacort, ... Read more

January 28, 2019February 12, 2019

Phase 2b Trial Testing Vamorolone Now Enrolling Boys Ages 4-6 with DMD

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A Phase 2b trial of the investigational therapy vamorolone is 4-to-6-year-old boys with Duchenne muscular dystrophy (DMD) has begun to enroll patients. Patient recruitment for the international ReveraGen BioPharma-sponsored, double-blind ... Read more

January 25, 2019February 13, 2019

Connecticut Man With Limb-girdle MD Inspires Others

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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 ... Read more

January 23, 2019January 23, 2019

PPMD Publishes 10-Year Duchenne and Becker Patient Registry Report

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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 ... Read more

January 21, 2019January 21, 2019

New Tool to Help With Virtual Research and Care Management for Duchenne Patients

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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 ... Read more

January 18, 2019January 18, 2019

Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program

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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 ... Read more

January 16, 2019January 16, 2019

Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

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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 ... Read more

January 14, 2019January 14, 2019

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

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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 ... Read more

January 11, 2019January 11, 2019

PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies

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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 ... Read more

January 9, 2019January 9, 2019

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

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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 ... Read more

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