Experimental Therapeutics of Neuromuscular Disease

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DESCRIPTION (provided by applicant): The conference will bring together translational and clinical scientists in a forum for discussion of current and novel therapies and draw speakers and participants from international experts with an interest in developing clinical trials. This proposal has been developed by a Steering Committee which includes the leadership of the Muscle Study Group (MSG) and the European TREAT-NMD consortia. The conference is open to attendance from these and other groups and by individuals not currently affiliated with a study group. It has a major focus on trainees and others interested in participating in the experimental therapeutics of neuromuscular disease. This international conference will be held in an informal, isolated environment 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 state-of-the-air presentations with a translational focus by laboratory and clinical investigators whose work is in/may be brought into clinical trial. Proposed speakers include Kate Bushby, Hanns Lochmuller, Jerry Mendell, John Day, Thurman Wheeler, Bertrand Fontaine, Michael Hanna and Stephen Cannon. (2) To discuss/debate the most appropriate and efficient ways to demonstrate clinical efficacy of novel treatment strategies. (3) To provide an informal environment away from competing activities for the interaction/development of collaboration between established investigators and between senior and new/junior investigators. (4) To give new investigators a sense of the "history" and longitudinal development of experimental therapeutics of neuromuscular disease by having senior "icons" of the field speak and interact with other participants: Michael Brooke, Andrew Engel, Lord John Walton of Denchant, Lewis P. Rowland. (5) To provide a collaborative environment for new project development among investigators. This is an application for funds for an international conference for scientists to discuss ways to conduct studies of new treatments for neuromuscular diseases, to exchange information about the causes of the diseases, and for senior scientists to educate junior scientists.