Duchenne Muscular Dystrophy (DMD)

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).

DMD/BMD: Questions About Ataluren’s Mechanism

It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.

DMD: Drisapersen Receives ‘Breakthrough Therapy’ Designation

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

DMD: Eteplirsen Shows Continued Benefit at 84 Weeks

Eteplirsen, an experimental exon-skipping drug designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 84 of a phase 2b, 12-person study.

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