Treatment Strategies for Neuromuscular Diseases: The Challenge of Recruitment

描述(申请者提供)：本申请提出一个国际会议：“神经肌肉疾病的治疗策略：招募的挑战”。这次会议将汇集来自学术界、基金会和制药行业的专家翻译和临床科学家在一个合议性论坛上。这项建议是由一个指导委员会制定的，该委员会包括翻译临床科学家和肌肉研究小组的领导。该会议向它和其他团体以及目前没有隶属于学习小组的个人开放。这次国际会议将在非正式、孤立的环境中举行，将把高级调查人员、制药公司的代表、NIH项目工作人员、基金会和患者倡导代表以及初级/见习调查人员聚集在一起。会议将涵盖从坐床到床边的一系列主题，目的是(1)讨论患有罕见神经肌肉疾病的志愿者的有效招募战略；(2)探讨从神经肌肉研究起步的招募和指导年轻神经科医生的有效方法；(3)为初级教员提供关于开发神经肌肉疾病新治疗策略的策略的培训；(4)由实验室和临床研究人员提供最先进的演示，重点是翻译，他们的工作正被纳入临床试验；(5)促进现有研究人员之间以及高级和新/初级研究人员之间的互动/发展协作。预计研讨会可能导致不同从属关系的与会者之间进行新的合作。会议的科学重点将集中在(1)炎症性肌肉疾病的基础和实验治疗方面，以及(2)将基因治疗引入临床试验的战略。受训者将被邀请提交他们的作品，并参加一个互动海报讨论环节。 与公共卫生相关：这是一次国际会议的资金申请，来自学术界、基金会和工业界的科学家将讨论如何合作开发和进行神经肌肉疾病的新疗法研究，交换有关疾病原因的信息，招募试验志愿者，并招募新的初级科学家来开发和进行此类试验。

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.