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治疗方法，并吸引对发展临床试验感兴趣的国际专家的演讲者和参与者。该建议是由一个指导委员会制定的，该委员会包括肌肉研究小组（MSG）和欧洲治疗- nmd联盟的领导。会议向这些团体和其他团体以及目前没有隶属于某个研究小组的个人开放。它主要关注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初级临床/转化研究人员的职业生涯。它将把高级研究人员、NIH项目人员和基金会代表与初级/实习研究人员聚集在一起。会议将涵盖一系列的主题，从实验室到床边的肌肉疾病。会议的目的是：(1)由实验室和临床研究人员提供具有翻译重点的最新报告，他们的工作正在或可能进入临床试验。提议的演讲者包括凯特·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/辩论最合适和有效的方法来证明新的治疗策略的临床疗效。(3)提供一个非正式的环境，避免竞争活动，促进现有调查人员之间、高级调查人员与新调查人员/初级调查人员之间的互动/合作发展。(4)通过邀请该领域的资深“代表人物”发言并与其他参与者互动，让新研究者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、邓钱特的约翰·沃尔顿勋爵、刘易斯·p·罗兰。(5)为研究人员之间的新项目开发提供协作环境。这是一份为科学家们讨论神经肌肉疾病新疗法研究方法的国际会议基金申请，交换有关疾病原因的信息，并为高级科学家教育初级科学家。

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.