Novel Designs and Outcome Measures for Bench to Bedside Research on NMD

NMD 从实验室到临床研究的新颖设计和成果衡量

• 批准号: 7406266
• 负责人: Robert C Griggs
• 金额: $ 2.94万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-09-05 至 2008-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7406266
• 关键词: Arts; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Development; Disease; Environment; Foundations; Funding; Generations; Goals; International; Learning; Mentors; Methodology; Molecular; Myopathy; Neuromuscular Diseases; Numbers; Outcome Measure; Patients; Public Health; Range; Research; Research Personnel; Research Training; Scientist; Sodium Glutamate; Therapeutic; United States National Institutes of Health; Work; bench to bedside; career; design; innovation; interest; novel; programs; symposium; translational approach

DESCRIPTION (provided by applicant): This application proposes an international research and training seminar to bring together senior clinical/translational investigators with trainees who share a common interest in developing new treatments for neuromuscular disease. These disorders pose special challenges in terms of developing outcome measures and designing trials for small numbers of patients. Molecular discoveries of the causes of most neuromuscular diseases have suggested new treatment strategies. It is timely and important to consider translational approaches to bringing molecular discoveries into well-designed clinical studies. This international conference will be held in an informal, isolated environment in 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 a program of: translational, bench-to-bedside presentations on neuromuscular diseases: scientific presentations on molecular neuromuscular disorders; innovative clinical trial methodology pertinent to uncommon diseases; and perspective on current funded studies in neuromuscular disease. (2) To provide opportunity for new trainees to learn about research in neuromuscular diseases and to become involved with the MSG and other clinical investigators. (3) To provide educational/scientific interactions among senior and junior investigators and trainees within a framework of successful, funded, investigative programs. (4) Provide a mentoring environment for idea generation and career development. (5) Provide "state-of-the-art" information and overview of neuromuscular experimental therapeutics. This international conference will bring together scientists to discuss ways to conduct studies of new treatments for neuromuscular diseases, new information about the molecular causes of the diseases, and ways for investigators to work together. In this effort to provide new treatments for neuromuscular disease, this conference will help to further public health goals.

描述（由申请人提供）：本申请提议举办一次国际研究和培训研讨会，将高级临床/转化研究人员与对开发神经肌肉疾病新疗法有共同兴趣的学员聚集在一起。这些疾病在制定结果指标和为少数患者设计试验方面提出了特殊的挑战。大多数神经肌肉疾病病因的分子发现提出了新的治疗策略。考虑将分子发现引入精心设计的临床研究的转化方法是及时且重要的。这次国际会议将在非正式、孤立的环境中举行，旨在扩大专业知识并指导临床/转化研究人员的职业生涯。它将把高级研究人员、NIH 项目工作人员、基金会代表以及初级/实习研究人员聚集在一起。会议将涵盖肌肉疾病从实验室到临床的一系列主题。会议的目标是： (1) 提供以下计划：关于神经肌肉疾病的转化性、从实验室到临床的演示：关于分子神经肌肉疾病的科学演示；与罕见疾病相关的创新临床试验方法；以及对当前资助的神经肌肉疾病研究的看法。 (2) 为新学员提供了解神经肌肉疾病研究并参与 MSG 和其他临床研究人员的机会。 (3) 在成功的、受资助的调查计划框架内，为高级和初级研究人员以及受训人员提供教育/科学互动。 (4) 为创意产生和职业发展提供指导环境。 (5) 提供神经肌肉实验疗法的“最新”信息和概述。这次国际会议将汇集科学家，讨论神经肌肉疾病新疗法的研究方法、有关疾病分子原因的新信息以及研究人员的合作方式。为了为神经肌肉疾病提供新的治疗方法，本次会议将有助于进一步实现公共卫生目标。