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）：本申请提出了一项国际研究和培训研讨会，将高级临床/翻译调查员与受训者结合在一起，他们在开发新的神经肌肉疾病治疗方面具有共同的兴趣。这些疾病在制定结果指标和设计少数患者的试验方面面临着特殊的挑战。大多数神经肌肉疾病的原因的分子发现提出了新的治疗策略。考虑转化方法将分子发现带入精心设计的临床研究是及时和重要的。该国际会议将在非正式的孤立环境中举行，旨在扩大专业知识并指导开始临床/转化调查员的职业。它将将高级调查员，NIH计划人员和基金会代表与初级/受训者调查员一起。该会议将涵盖从肌肉疾病中的基准阶层进行的一系列主题。会议的目的是：（1）提供：关于神经肌肉疾病的转化，基准对床的计划：分子神经肌肉疾病的科学演示；与罕见疾病有关的创新临床试验方法；以及对当前基金会疾病的资助研究的观点。 （2）为新学员提供了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，资助的调查计划的框架内提供高级和初级研究人员和学员之间的教育/科学互动。 （4）为思想产生和职业发展提供了指导环境。 （5）提供“最新”信息和神经肌肉实验疗法的概述。这次国际会议将召集科学家讨论如何进行神经肌肉疾病的新治疗方法，有关疾病分子原因的新信息以及研究人员共同工作的方法。为了为神经肌肉疾病提供新的治疗方法，该会议将有助于进一步的公共卫生目标。