Novel treatment for muscle disease: Fueling the pipeline and finding the product

描述(由申请人提供)：在最近一篇关于“翻译和临床科学”的文章中，NIH主任Elias Zerhouni指出，“基础科学家和翻译科学家之间需要强有力的双向信息流……”肌肉研究小组是一个由临床和翻译研究人员组成的合作小组，他们的研究得到了美国国立卫生研究院和其他外部资金的支持，目的是：“促进对肌肉疾病和相关神经肌肉疾病的病因(S)、发病机制、流行病学和临床表现的了解；制定和实施策略，以检查有希望的治疗干预措施。”这项提案要求支持为期两天的临床科学家会议，他们共同关注开发神经肌肉疾病的新疗法。会议将把初级调查员和受训人员聚集在一起，加入资深资深调查员的行列。参与者代表美国、加拿大和欧洲的主要医疗中心、行业以及NIH和FDA。科学计划将强调转化性和实验性治疗学研究。所要求的支助将使目前没有经费的受训人员和其他初级调查人员能够出席和参加会议。会议由一个项目委员会计划，包括两个主要会议：(1)关于肌营养不良症、神经肌肉连接障碍和运动神经元疾病等主题的床边翻译陈述；(2)包括行业、FDA和学术观点的临床试验方法学会议。会议将在肌肉研究小组会议的框架内举行，该会议作为审查外部资助临床试验的进展和计划的论坛。所有与会者，包括实习生和初级教员，都有机会参与这些试验的讨论，使他们对当前广泛的临床/转化性研究有一个横截面的观点。这次会议还将提供一个非正式的环境，以(1)促进教育和科学互动；(2)促进致力于开发神经肌肉疾病新疗法的知名研究人员之间的合作；(3)提供一个框架，促进现有研究人员和初级研究人员之间的合作；(4)为创意的产生和职业发展提供指导环境。这次会议很重要，因为它将帮助高级和初级研究人员更多地了解神经肌肉疾病研究中适当的转译研究和设计，并发展开展临床研究的合作。

DESCRIPTION (provided by applicant): In a recent article on "Translational and clinical science", NIH Director Elias Zerhouni noted "the need for a robust, bidirectional information flow between basic and translational scientists...". The Muscle Study Group is a collaboration of clinical and translational investigators whose research is supported by NIH and other extramural funding with the aims of: "advancing knowledge about the cause(s), pathogenesis, epidemiology, and clinical manifestations of muscle disease and related neuromuscular disorders; developing and implementing strategies to examine promising therapeutic interventions." This proposal requests support for a 2-day meeting of clinical scientists who share a common focus on developing novel treatments for neuromuscular disease. The meeting will bring together junior investigators and trainees to join senior, established investigators. Participants represent major medical centers in the U.S., Canada, and Europe, industry and the NIH and FDA. The scientific program will emphasize translational and experimental therapeutics research. The support requested will make it possible for currently-unfunded trainees and other junior investigators to attend and participate in the meeting. The meeting has been planned by a Program Committee and includes two major sessions: (1) translational bench-to-bedside presentations on topics including muscular dystrophy, neuromuscular junction disorders and motor neuron disorders; (2) clinical trial methodology session including industry, FDA and academic perspectives. The meeting will take place within the framework of a Muscle Study Group meeting that serves as a forum for reviewing the progress and plans for extramurally-funded clinical trials. All meeting participants including trainees and junior faculty have the opportunity to participate in discussions of these trials giving them a cross-sectional perspective on a wide range of current clinical/translational research. This meeting will also provide an informal setting that (1) promotes educational and scientific interactions; (2) fosters collegiality among established investigators committed to developing novel treatments for neuromuscular disease; (3) provides a framework for developing collaborations between established and junior investigators; (4) provides a mentoring environment for idea generation and career development. This meeting is important because it will help both senior and junior investigators to learn more about appropriate translational research and design in the study of neuromuscular disease and to develop collaborations to carry out the clinical research.