Novel treatment for muscle disease: Fueling the pipeline and finding the product

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DESCRIPTION (provided by applicant): In a recent article on "Translational and clinical science", NIH Director Elias Zerhouni noted "the need for a robust, bidirectional information flow between basic and translational scientists...". The Muscle Study Group is a collaboration of clinical and translational investigators whose research is supported by NIH and other extramural funding with the aims of: "advancing knowledge about the cause(s), pathogenesis, epidemiology, and clinical manifestations of muscle disease and related neuromuscular disorders; developing and implementing strategies to examine promising therapeutic interventions." This proposal requests support for a 2-day meeting of clinical scientists who share a common focus on developing novel treatments for neuromuscular disease. The meeting will bring together junior investigators and trainees to join senior, established investigators. Participants represent major medical centers in the U.S., Canada, and Europe, industry and the NIH and FDA. The scientific program will emphasize translational and experimental therapeutics research. The support requested will make it possible for currently-unfunded trainees and other junior investigators to attend and participate in the meeting. The meeting has been planned by a Program Committee and includes two major sessions: (1) translational bench-to-bedside presentations on topics including muscular dystrophy, neuromuscular junction disorders and motor neuron disorders; (2) clinical trial methodology session including industry, FDA and academic perspectives. The meeting will take place within the framework of a Muscle Study Group meeting that serves as a forum for reviewing the progress and plans for extramurally-funded clinical trials. All meeting participants including trainees and junior faculty have the opportunity to participate in discussions of these trials giving them a cross-sectional perspective on a wide range of current clinical/translational research. This meeting will also provide an informal setting that (1) promotes educational and scientific interactions; (2) fosters collegiality among established investigators committed to developing novel treatments for neuromuscular disease; (3) provides a framework for developing collaborations between established and junior investigators; (4) provides a mentoring environment for idea generation and career development. This meeting is important because it will help both senior and junior investigators to learn more about appropriate translational research and design in the study of neuromuscular disease and to develop collaborations to carry out the clinical research.