Experimental Therapeutics of Neuromuscular Disease

描述（由申请人提供）：会议将汇集翻译和临床科学家在一个论坛上讨论当前和新的疗法，并吸引演讲者和参与者从国际专家与开发临床试验的兴趣。这项建议是由一个指导委员会拟订的，该委员会包括肌肉研究小组和欧洲反弹道导弹-国家导弹防御系统联合会的领导人。会议对这些团体和其他团体以及目前不属于研究小组的个人开放。它主要侧重于学员和其他有兴趣参与神经肌肉疾病实验治疗的人。本次国际会议将在一个非正式的，孤立的环境中举行，旨在扩大专业知识，并指导开始临床/翻译研究者的职业生涯。它将把高级研究人员、NIH项目工作人员和基金会代表与初级/实习研究人员聚集在一起。会议将涵盖从肌肉疾病的板凳到床边的一系列主题。会议的目的是：（1）由实验室和临床研究者提供最新的演讲，其工作正在/可能被带入临床试验。拟议的发言人包括凯特布什比，汉斯洛克穆勒，杰里门德尔，约翰戴，瑟曼惠勒，伯特兰方丹，迈克尔汉纳和斯蒂芬坎农。(2)讨论/辩论证明新型治疗策略临床疗效的最适当和最有效的方法。(3)提供一个非正式的环境，使常设调查员之间以及高级和新/初级调查员之间能够相互交流/发展合作，而不参与相互竞争的活动。(4)通过让该领域的高级“偶像”发言并与其他参与者互动，让新研究人员了解神经肌肉疾病实验疗法的“历史”和纵向发展：迈克尔布鲁克，安德鲁恩格尔，登尚的约翰沃尔顿勋爵，刘易斯P.罗兰。(5)为研究者之间的新项目开发提供协作环境。这是一份国际会议资金申请，供科学家讨论如何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，以及资深科学家教育年轻科学家。

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.