Treatment Strategies for Neuromuscular Diseases: The Challenge of Recruitment

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DESCRIPTION (provided by applicant): This application proposes an international conference: "Treatment Strategies for Neuromuscular Diseases: The Challenge of Recruitment". 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. 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 to (1) discuss effective recruitment strategies for volunteers with rare neuromuscular diseases; (2) look at effective methods of recruiting and mentoring young neurologists starting out in neuromuscular research (3) provide training for junior faculty in the strategies for developing novel treatment strategies for neuromuscular disease; (4) offer state-of-the-art presentations with a translational focus by laboratory and clinical investigators whose work is being brought into clinical trial; (5) provide for the 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. The conference's scientific emphases will be on (1) fundamental and experimental therapeutic aspects of inflammatory muscle diseases and (2) strategies for bringing gene therapy into clinical trials. Trainees 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 to develop and conduct studies of new treatments for neuromuscular diseases, to exchange information about the causes of the diseases, to recruit volunteers for the trials, and to recruit new junior scientists to develop and conduct such trials.