Translational Neuromuscular Research, Diverse Diseases, Convergent Themes

描述（由申请人提供）：本申请提出了一个国际会议：“转化神经肌肉研究：多样的疾病，会聚的主题”。会议将汇集来自学术界，基金会和制药公司的专家翻译和临床科学家参加一个合议论坛。该提案由指导委员会制定，该委员会包括翻译临床科学家和肌肉研究小组的领导。会议对本组织和其他团体以及目前不属于研究小组的个人开放。会议的科学重点将是一些关键的“交叉”主题，这些主题对多种神经肌肉疾病是共同的，并具有重要的转化潜力。这次国际会议将在一个非正式的，孤立的环境中举行，将使高级研究人员，制药公司的代表，NIH项目工作人员，基金会和患者倡导代表与初级/实习研究人员聚集在一起。是次研讨会的主题涵盖从临床到临床的多个范畴，旨在（1）探讨RNA代谢在不同神经肌肉疾病中的发病机制及潜在治疗靶点;（2）探讨动物模型在神经肌肉疾病转化研究中的作用;（3）讨论运动对神经肌肉疾病的治疗作用;（4）探讨运动对神经肌肉疾病的影响。（4）为初级教员提供培训，以制定神经肌肉疾病的新治疗策略;（5）为现有研究人员以及高级和新/初级研究人员之间的互动/合作发展提供支持。预计研讨会可能会导致各种附属机构的与会者之间的新的合作。将邀请与会者提交他们的工作，并参加互动海报讨论会。 公共卫生相关性：这是一个国际会议的资金申请，来自学术界，基金会和工业界的科学家讨论如何共同努力，进行神经肌肉疾病新疗法的研究，交流有关疾病原因的信息，并招募新的年轻科学家来开发和进行此类试验。

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.