Duchenne Muscular Dystrophy (DMD)

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.

DMD — Hansell Stedman, M.D.

Hansell Stedman, associate professor of surgery at the University of Pennsylvania in Philadelphia, was awarded an MDA research grant totaling $300,000 over a period of three years to study early immune responses during administration of gene therapy.

DMD — Douglas Millay, Ph.D.

Douglas Millay, a postdoctoral researcher at the University of Texas Southwestern Medical Center in Dallas, was awarded an MDA development grant totaling $180,000 over a period of three years to study the process of myoblast fusion.

DMD/BMD — Lynn Megeney, Ph.D.

Lynn Megeney, senior scientist at the Sprott Centre for Stem Cell Research, Ottawa Hospital Research Institute in Ottawa, Ontario, Canada, was awarded an MDA research grant totaling $300,000 over a period of three years to study how muscle stem cells are controlled.

DMD — Charles Gersbach, Ph.D.

Charles Gersbach, assistant professor at Duke University in Durham, N.C., was awarded an MDA research grant totaling $300,000 over a period of three years to develop a new approach for gene therapy in Duchenne muscular dystrophy (DMD).

DMD — Moon-Chang Choi, Ph.D.

Moon-Chang Choi, a postdoctoral researcher at Duke University in Durham, N.C., was awarded an MDA development grant totaling $120,000 over a period of two years to study one of the molecular controls regulating muscle repair.

MDA Statement RE: Sarepta Therapeutics To Submit NDA for Duchenne Muscular Dystrophy Next Year

DMD: Sarepta to Submit New Drug Application to FDA for Eteplirsen in 2014

 In a July 24, 2013, press release and conference call, biotechnology company Sarepta Therapeutics provided an update on the regulatory pathway for eteplirsen, the company’s experimental drug for Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin

DMD — ReveraGen BioPharma

The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular dystrophy (DMD).

DMD — Catabasis Pharmaceuticals

The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals. The award is part of a strategic partnership under which the pharmaceutical company will test two compounds — CAT-1004 and CAT-1040 — in the mdx research mouse model of Duchenne muscular dystrophy (DMD).

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