Translational Neuromuscular Research, Diverse Diseases, Convergent Themes

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DESCRIPTION (provided by applicant): This application proposes an international conference: "Translational Neuromuscular Research: Diverse Diseases, Convergent Themes". The conference will bring together expert translational and clinical scientists from academia, foundations, and pharma in a collegial forum. This proposal has been developed by a Steering Committee that includes both translational clinical scientists and leadership of the Muscle Study Group. The conference is open to attendance from it and other groups and by individuals not currently affiliated with a study group. The conference's scientific emphasis will be on a number of key "cross cutting" themes that are common to multiple neuromuscular disorders and have important translational potential. This international conference, to be held in an informal, isolated environment, will bring senior investigators, representatives of pharma, NIH program staff, and foundation and patient advocacy representatives together with junior/trainee investigators. The conference will cover a range of topics from bench-to-bedside in muscle disease with the aims (1) to explore the role of RNA metabolism as disease mechanism and as a potential therapeutic target in different neuromuscular diseases; (2) to examine the role of animal models in translational research in neuromuscular disease; (3) to discuss exercise as a therapy in neuromuscular diseases; (4) to provide training for junior faculty in the strategies for developing novel treatment strategies for neuromuscular disease; (5) to provide for interaction/development of collaboration among established investigators and between senior and new/junior investigators. It is expected that the seminar may lead to new collaboration among attendees of various affiliations. Attendees will be invited to submit their work and to participate in an interactive poster discussion session. PUBLIC HEALTH RELEVANCE: This is an application for funds for an international conference for scientists from academia, foundations, and industry to discuss ways to work together and conduct studies of new treatments for neuromuscular diseases, to exchange information about the causes of the diseases, and to recruit new junior scientists to develop and conduct such trials.