Duchenne Muscular Dystrophy (DMD)

DMD - Da-Zhi Wang, Ph.D.

Da-Zhi Wang, an associate professor of pediatrics at Boston Children's Hospital and Harvard Medical School, was awarded an MDA research grant totaling $253,800 over three years to study the role of a compound called miR-155, a "microRNA," in muscle function and regeneration and to see whether reducing it could help treat Duchenne muscular dystrophy (DMD). Wang has found that mice with lacking miR-155 have better muscle function and regeneration than mice that have this substance.

DMD - Terence Partridge, Ph.D.

Terence Partridge, a professor at Children’s Research Institute at Children’s National Medical Center in Washington, D.C., was awarded an MDA research grant totaling $253,800 over three years to develop and test a new type of compound that causes exon skipping – a way for cells to make functional proteins from flawed genes -- as a potential treatment for Duchenne muscular dystrophy (DMD).  Current exon-skipping drugs for DMD have limited, if any, ability to enter heart muscle cells.

DMD - Erica Reeves, Ph.D.

Erica Reeves, a biologist and vice president of research and operations at ReveraGen BioPharma, was awarded an MDA translational research grant totaling of $1,015,200 over one year to conduct the first human trial of VBP15, an experimental drug in development  to treat Duchenne muscular dystrophy (DMD).  This first trial will be in healthy volunteers.

Drug Development for DMD: Fall 2014 Update

Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.

PTC is moving forward with ataluren

Sarepta Announces Revised Timeline for Eteplirsen FDA Application

Boys with Duchenne muscular dystrophy between the ages of seven and 16 are now being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.

DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

Cambridge, Mass.-based biotechnology company Sarepta Therapeutics updated the Duchenne muscular dystrophy (DMD) community about its pipeline of drugs in development for this disorder via an Aug.

First Drug for Duchenne Muscular Dystrophy Gets 'Conditional Approval' in Europe

DMD: Ataluren Receives Conditional Approval in Europe

Update (Oct. 24, 2014): Enrollment for the phase 3 trial of ataluren is complete, with results expected in the second half of 2015. See the PTC press release of Sept. 9, 2014.

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original story:

PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ for the treatment of Nonsense Mutation Duchenne Muscular Dystrophy

DMD: ReveraGen Drug Will Move to Human Testing

ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.

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