Treatment Strategies for Neuromuscular Diseases: The Challenge of Recruitment

神经肌肉疾病的治疗策略：招募的挑战

• 批准号: 8004626
• 负责人: Robert C Griggs
• 金额: $ 4.49万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-08-01 至 2011-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8004626
• 关键词: Academia; Arts; Clinical; Clinical Investigator; Clinical Trials; Collaborations; Development; Disease; Environment; Faculty; Foundations; Funding; Individual; Industry; International; Laboratories; Lead; Leadership; Mentors; Methods; Muscle; Myopathy; Myositis; Neurologist; Neuromuscular Diseases; Patient advocacy; Recruitment Activity; Research; Research Personnel; Scientist; Therapeutic; Training; United States National Institutes of Health; Work; bench to bedside; gene therapy; neuromuscular; novel; posters; programs; public health relevance; symposium; treatment strategy; volunteer

DESCRIPTION (provided by applicant): This application proposes an international conference: "Treatment Strategies for Neuromuscular Diseases: The Challenge of Recruitment". The conference will bring together expert translational and clinical scientists from academia, foundations, and pharma in a collegial forum. This proposal has been developed by a Steering Committee that includes both translational clinical scientists and leadership of the Muscle Study Group. The conference is open to attendance from it and other groups and by individuals not currently affiliated with a study group. This international conference, to be held in an informal, isolated environment, will bring senior investigators, representatives of pharma, NIH program staff, and foundation and patient advocacy representatives together with junior/trainee investigators. The conference will cover a range of topics from bench-to-bedside in muscle disease with the aims to (1) discuss effective recruitment strategies for volunteers with rare neuromuscular diseases; (2) look at effective methods of recruiting and mentoring young neurologists starting out in neuromuscular research (3) provide training for junior faculty in the strategies for developing novel treatment strategies for neuromuscular disease; (4) offer state-of-the-art presentations with a translational focus by laboratory and clinical investigators whose work is being brought into clinical trial; (5) provide for the interaction/development of collaboration among established investigators and between senior and new/junior investigators. It is expected that the seminar may lead to new collaboration among attendees of various affiliations. The conference's scientific emphases will be on (1) fundamental and experimental therapeutic aspects of inflammatory muscle diseases and (2) strategies for bringing gene therapy into clinical trials. Trainees will be invited to submit their work and to participate in an interactive poster discussion session. PUBLIC HEALTH RELEVANCE: This is an application for funds for an international conference for scientists from academia, foundations, and industry to discuss ways to work together to develop and conduct studies of new treatments for neuromuscular diseases, to exchange information about the causes of the diseases, to recruit volunteers for the trials, and to recruit new junior scientists to develop and conduct such trials.

描述（由申请人提供）：本申请提议召开一次国际会议：“神经肌肉疾病的治疗策略：招募的挑战”。会议将汇集来自学术界、基金会和制药界的专家转化和临床科学家，参加一个学术论坛。该提案由指导委员会制定，该委员会包括转化临床科学家和肌肉研究组的领导层。该会议向本研究组和其他团体以及目前不属于研究组的个人开放。这次国际会议将在非正式、孤立的环境中举行，高级研究人员、制药公司代表、NIH 项目工作人员、基金会和患者倡导代表以及初级/实习研究人员将齐聚一堂。会议将涵盖肌肉疾病从实验室到临床的一系列主题，目的是（1）讨论罕见神经肌肉疾病志愿者的有效招募策略； (2) 研究招募和指导从事神经肌肉研究的年轻神经科医生的有效方法 (3) 为初级教师提供开发神经肌肉疾病新治疗策略的培训； (4) 由实验室和临床研究人员提供最先进的演讲，重点关注其工作正在被带入临床试验的转化； (5) 规定现有研究者之间以及高级和新/初级研究者之间的互动/发展合作。预计研讨会可能会导致不同背景的与会者之间建立新的合作。会议的科学重点将是（1）炎症性肌肉疾病的基础和实验治疗方面以及（2）将基因疗法引入临床试验的策略。学员将被邀请提交他们的作品并参加互动海报讨论会。 公共卫生相关性：这是为来自学术界、基金会和工业界的科学家举办国际会议申请资金，以讨论如何共同努力开发和开展神经肌肉疾病新疗法的研究，交流有关疾病原因的信息，招募试验志愿者，以及招募新的初级科学家来开发和进行此类试验。