Translational Neuromuscular Research, Diverse Diseases, Convergent Themes

描述（由申请人提供）：本申请申请举办国际会议：“转化神经肌肉研究：多样疾病，融合主题”。会议将汇集来自学术界、基金会和制药公司的专家转化和临床科学家。该建议是由指导委员会制定的，该委员会包括转化临床科学家和肌肉研究小组的领导。会议开放供它和其他小组以及目前没有隶属于某一研究小组的个人出席。会议的科学重点将放在一些关键的“交叉”主题上，这些主题是多种神经肌肉疾病所共有的，并且具有重要的转化潜力。这次国际会议将在非正式的、孤立的环境中举行，将汇集高级研究人员、制药公司代表、NIH项目人员、基金会和患者权益代表以及初级/实习研究人员。会议将涵盖从实验室到床边的一系列主题，其目的是：(1)探索RNA代谢作为疾病机制的作用，并作为不同神经肌肉疾病的潜在治疗靶点；(2)研究动物模型在神经肌肉疾病转化研究中的作用；(3)探讨运动作为神经肌肉疾病的治疗方法；(4)为初级教员提供开发神经肌肉疾病新治疗策略的培训；(5)为既有调查人员之间，以及高级调查人员与新调查人员/初级调查人员之间提供互动/发展合作的机会。预计这次研讨会可能会导致不同背景的与会者之间的新的合作。与会者将被邀请提交他们的作品，并参加一个互动的海报讨论环节。

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.