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”。该会议将在一个大学论坛上汇集来自学术界，基金会和制药的专家翻译和临床科学家。该提案是由指导委员会制定的，其中包括转化临床科学家和肌肉研究小组的领导。该会议开放，并由目前尚未与研究小组相关的个人以及其他团体的出席率开放。这次国际会议将在非正式的，孤立的环境中举行，将带来高级调查员，制药公司，NIH计划人员以及基金会和患者倡导代表以及初级/受训者调查员。该会议将涵盖从肌肉疾病中的基准阶层到卧铺的一系列主题，目的是（1）讨论罕见神经肌肉疾病的志愿者的有效招募策略； （2）研究招募和指导年轻神经病学家的有效方法，从神经肌肉研究开始（3）为初级教师提供培训，以制定神经肌肉疾病的新型治疗策略的策略； （4）提供最先进的演讲，并由实验室和临床研究人员的翻译重点进行，他们的工作正在临床试验中； （5）规定了知名研究人员以及高级和初级调查人员之间合作的互动/开发。预计该研讨会可能会导致各种分支机构的参与者之间的新合作。会议的科学重点将放在（1）炎症性肌肉疾病的基本和实验性治疗方面以及（2）将基因治疗带入临床试验中的策略。将邀请学员提交工作，并参加互动海报讨论会议。 公共卫生相关性：这是针对来自学术界，基金会和行业的科学家的国际会议申请资金的申请，以讨论如何共同开发和开发神经肌肉疾病的新治疗方法的方法，以交换有关疾病原因的信息，以招募志愿者招募志愿者，以招募新的初级科学家开发和进行此类试验。