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

肌肉疾病的新疗法：为管道加油并寻找产品

• 批准号: 7160327
• 负责人: Robert C Griggs
• 金额: $ 2.8万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-06-09 至 2007-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7160327
• 关键词: clinical trials; degenerative motor system disease; experimental designs; meeting /conference /symposium; muscular dystrophy; musculoskeletal disorder therapy; nervous system disorder therapy; neuromuscular disorder; travel

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.

描述（由申请人提供）：在最近一篇关于“转化和临床科学”的文章中，NIH 主任 Elias Zerhouni 指出“基础科学家和转化科学家之间需要一个强大的、双向的信息流……”。 肌肉研究小组是一个由临床和转化研究人员组成的合作组织，其研究得到 NIH 和其他校外资金的支持，其目的是：“增进对肌肉疾病和相关神经肌肉疾病的病因、发病机制、流行病学和临床表现的了解。疾病；制定和实施策略来检查有希望的治疗干预措施。”该提案请求支持为期 2 天的临床科学家会议，他们的共同重点是开发神经肌肉疾病的新疗法。 该会议将汇集初级调查员和实习生，以加入高级、成熟的调查员行列。 参与者代表美国、加拿大和欧洲的主要医疗中心、工业界以及 NIH 和 FDA。 该科学计划将强调转化和实验疗法研究。 所请求的支持将使目前没有资金的受训人员和其他初级调查人员能够出席和参加会议。 本次会议由计划委员会策划，包括两个主要会议：（1）从实验室到临床的转化演讲，主题包括肌营养不良症、神经肌肉接头疾病和运动神经元疾病； (2)临床试验方法论会议，包括行业、FDA和学术观点。 该会议将在肌肉研究组会议的框架内举行，该会议将作为审查外部资助的临床试验的进展和计划的论坛。 所有会议参与者，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/转化研究有一个横断面的视角。 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，以发展资深研究者和初级研究者之间的合作； (4)为创意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化研究和设计，并开展合作来开展临床研究。