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）：本申请提出了一个国际研究和培训研讨会，将高级临床/翻译研究人员与在开发神经肌肉疾病新疗法方面有共同兴趣的受训人员聚集在一起。这些疾病在为少数患者制定结局指标和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。考虑将分子发现转化为精心设计的临床研究是及时和重要的。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）提供一个程序：神经肌肉疾病的翻译，从实验室到床边的介绍;分子神经肌肉疾病的科学介绍;与罕见疾病相关的创新临床试验方法;以及对目前资助的神经肌肉疾病研究的看法。(2)为新学员提供学习神经肌肉疾病研究的机会，并参与MSG和其他临床研究人员的工作。(3)在成功的，资助的，调查方案的框架内，在高级和初级调查人员和受训人员之间提供教育/科学互动。(4)为创意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如何进行神经肌肉疾病的新疗法的研究，有关疾病的分子原因的新信息，以及研究人员合作的方式。在为神经肌肉疾病提供新疗法的努力中，本次会议将有助于进一步实现公共卫生目标。