Novel Designs and Outcome Measures for Bench to Bedside Research on NMD

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DESCRIPTION (provided by applicant): This application proposes an international research and training seminar to bring together senior clinical/translational investigators with trainees who share a common interest in developing new treatments for neuromuscular disease. These disorders pose special challenges in terms of developing outcome measures and designing trials for small numbers of patients. Molecular discoveries of the causes of most neuromuscular diseases have suggested new treatment strategies. It is timely and important to consider translational approaches to bringing molecular discoveries into well-designed clinical studies. This international conference will be held in an informal, isolated environment in designed to expand the expertise and to mentor the career of beginning clinical/translational investigators. It will bring senior investigators, NIH program staff, and foundation representatives together with junior/trainee investigators. The conference will cover a range of topics from bench-to-bedside in muscle disease. The aims of the conference are: (1) To provide a program of: translational, bench-to-bedside presentations on neuromuscular diseases: scientific presentations on molecular neuromuscular disorders; innovative clinical trial methodology pertinent to uncommon diseases; and perspective on current funded studies in neuromuscular disease. (2) To provide opportunity for new trainees to learn about research in neuromuscular diseases and to become involved with the MSG and other clinical investigators. (3) To provide educational/scientific interactions among senior and junior investigators and trainees within a framework of successful, funded, investigative programs. (4) Provide a mentoring environment for idea generation and career development. (5) Provide "state-of-the-art" information and overview of neuromuscular experimental therapeutics. This international conference will bring together scientists to discuss ways to conduct studies of new treatments for neuromuscular diseases, new information about the molecular causes of the diseases, and ways for investigators to work together. In this effort to provide new treatments for neuromuscular disease, this conference will help to further public health goals.