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）：本申请提出了一个国际会议：“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，基金会和制药公司的专家翻译和临床科学家参加一个合议论坛。该提案由指导委员会制定，该委员会包括翻译临床科学家和肌肉研究小组的领导。会议对本组织和其他团体以及目前不属于研究小组的个人开放。这次国际会议将在一个非正式的，孤立的环境中举行，将使高级研究人员，制药公司的代表，NIH项目工作人员，基金会和患者倡导代表与初级/实习研究人员聚集在一起。会议将涵盖从临床到临床的一系列肌肉疾病的主题，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）寻找招募和指导年轻神经学家开始神经肌肉研究的有效方法（3）为初级教师提供开发神经肌肉疾病新治疗策略的策略培训;（4）提供最先进的演示文稿，由实验室和临床研究者进行翻译，其工作将被纳入临床试验;（5）提供既定研究者之间以及高级和新/初级研究者之间的互动/合作发展。预计研讨会可能会导致各种附属机构的与会者之间的新的合作。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和（2）将基因治疗纳入临床试验的策略。将邀请受训人员提交他们的工作，并参加互动式海报讨论会。 公共卫生相关性：这是一份为来自学术界、基金会和工业界的科学家举办的国际会议的资金申请，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，并招募新的年轻科学家来开发和开展此类试验。