March 2015
Together we can Cure Duchenne!
Duchenne Research Highlights from MDA Conference

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The Muscular Dystrophy Association will hold its 2015 Scientific Conference at the Washington Marriott Wardman Park in Washington, D.C., March 11-14. The meeting is the preeminent gathering of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care, designed to accelerate discoveries and drug development for families fighting neuromuscular diseases, for which there are currently no cures. Here are some of the highlights on Duchenne research that was presented.

  • Edward Kaye from Sarepta Therapeutics presented his company’s most recent data about its experimental drug eteplirsen, designed to treat about 13 percent of the Duchenne muscular dystrophy (DMD) population, and discussed plans for development of additional drugs.
  • Joanne Donovan from Catabasis Pharmaceuticals discussed her company’s data about CAT-1004, an experimental anti-inflammatory drug in development to treat DMD regardless of the specific dystrophin gene mutation.
  • Mike Boss from Summit Therapeutics presented the latest information about his company’s experimental DMD compound SMT C1100. SMT C1100 is designed to sustain production of a protein known as utrophin in muscle fibers, potentially improving the health and sturdiness of these fibers and therefore prolonging muscle function.
  • Craig McDonald from the University of California, Davis, presented results from an eight-week, pilot trial of a compound called epicatechin in seven adults with Becker muscular dystrophy (BMD).

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