Duchenne Muscular Dystrophy (DMD)

Nationwide Children’s Podcast Explores Therapy Development for DMD

In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy

DMD — Anne Connolly, M.D.

Anne Connolly, a pediatric neurologist at Washington University in St. Louis, was awarded an MDA human clinical trial grant totaling $343,787 over a period of nearly three years (through April 30, 2016) to conduct a trial of prednisone in boys with Duchenne muscular dystrophy (DMD) who are younger than 2½ years old.

DMD — Morayma Reyes, M.D., Ph.D.

Morayma Reyes, assistant professor of pathology at the University of Washington in Seattle, was awarded an MDA research grant totaling $300,000 over a period of three years to study a strategy for reducing heart muscle damage in Duchenne muscular dystrophy (DMD).

DMD: Multicenter Trial To Test Drug that Fights Muscle Scarring, Inflammation

A phase 1b/2a clinical trial to test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 (also called delayed-release halofuginone) in boys with Duchenne muscular dystrophy (DMD) is now open at four sites in Maryland, Missouri and Ohio, with an additional site expected to open in California.

DMD/BMD — Terence Partridge, Ph.D.

Terence Partridge, professor of integrative systemic biology and pediatrics at George Washington University and associate director of the Children’s Research Institute in Washington, D.C., was awarded an MDA research grant totaling $300,000 over a period of three years to investigate differences in muscle repair mechanisms in mice and humans, in the context of Duchenne muscular dystrophy (DMD).

DMD — Rachelle Crosbie-Watson, Ph.D.

Rachelle Crosbie-Watson, professor of neurology at the University of California, Los Angeles, was awarded an MDA research grant totaling $300,000 over a period of three years to study whether increasing levels of the sarcospan protein can be therapeutic for Duchenne muscular dystrophy (DMD) and other muscle diseases.

DMD — Carmen Bertoni, Ph.D.

Carmen Bertoni, assistant professor of neurology at the University of California, Los Angeles, was awarded an MDA research grant totaling $300,000 over a period of three years to advance gene-editing strategies for Duchenne muscular dystrophy (DMD).

DMD — Steve Wilton, Ph.D.

Steve Wilton, foundation chair in molecular therapies at the Centre for Comparative Genomics at Murdoch (Australia) University, was awarded an MDA research grant totaling $300,000 over a period of three years to develop exon-skipping compounds for the less common mutations causing Duchenne muscular dystrophy (DMD).

DMD — Zejing Wang, M.D., Ph.D.

Zejing Wang, associate in clinical research at the Fred Hutchinson Cancer Research Center in Seattle, was awarded an MDA research grant totaling $300,000 over a period of three years to develop gene therapy for Duchenne muscular dystrophy (DMD) — with a focus on treating the heart — in a canine (dog) model of the disease.

Muscular Dystrophies — Lee Sweeney, Ph.D.

Lee Sweeney, director of the Penn Center for Orphan Disease Research and Therapy at the University of Pennsylvania Perelman School of Medicine in Philadelphia, was awarded an MDA research grant totaling $278,286 over a period of three years to test whether a new treatment that affects muscle calcium can slow the damage to muscle tissue in several forms of muscular dystrophy.

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