2013 International Research Conference on Tuberous Sclerosis Complex and Related

描述（由申请人提供）：本次国际会议的目标是促进合作研究，以解决结节性硬化症（TSC）和相关疾病（包括自闭症，癫痫，淋巴管平滑肌瘤病（LAM），癌症和具有重叠表型的罕见疾病）患者未满足的医疗需求。由于与TSC相关的症状种类繁多，对TSC的研究将增加与没有TSC的个体中发生的类似症状相关的知识。同样，在自闭症、癫痫和癌症等更广泛领域的进展可以提高对TSC的理解，从而改善TSC的治疗。这次会议是重要和及时的，部分原因是2012年6月举行的TSC临床共识会议更新了诊断，监测和管理TSC的建议。这些建议揭示了需要通过进一步研究加以解决的知识差距。基于以前的类似会议，2013年的研究会议将吸引超过200名与会者，包括大约20名特邀演讲者，10名从提交的摘要中选出的口头报告和30张海报。会议将使那些本来没有机会见面的人聚集在一起。除了如上所述邀请海训方案领域以外的演讲者外，组委会还将从提交的摘要中选出15名受训人员或初级研究人员，以获得旅行奖，以抵消交通、酒店和注册费用。在会议期间的星期六上午，TS联盟将在华盛顿举办第二届年度全国步行购物中心，为研究人员提供一个独特的机会，直接与受TSC影响的个人接触。本次会议保持了TS联盟主办的以往成功会议的结构和后勤特点，同时改变了主题，并以重要的方式邀请与会者。为了确保所提出的概念与以前的会议有很大的不同，2013年邀请的演讲者中有72%没有参加2011年的会议，44%根本没有参加2011年的会议。为了确保新的知识和想法被提出，22%的受邀演讲者在PubMed上没有或只有一篇关于“结节性硬化症”的文章。大多数会议包括非临床和临床演示，以鼓励思考和互动沿着连续的转化科学。没有同步平台会议，因此所有与会者都可以听到并讨论所有口头报告。30分钟的休息是为了鼓励网络。在两个用餐时间，小组讨论将使互动问答会议与来自不同规模的制药和生物技术公司的高级研究人员在一天，并与NCATS的高级领导人在另一天。各工作组将讨论和制定解决关键问题的行动。2013年会议是否成功，将根据执行工作组建议的影响以及通过会议期间讨论的合作项目加速研究的情况来衡量。

DESCRIPTION (provided by applicant): The goal of this international conference is to stimulate collaborative research to address unmet medical needs of those affected by tuberous sclerosis complex (TSC) and related disorders, including autism, epilepsy, lymphangioleiomyomatosis (LAM), cancer, and rare diseases with overlapping phenotypes. Because of the wide variety of symptoms associated with TSC, research into TSC will increase knowledge relevant to similar symptoms that occur in individuals without TSC. Likewise, advances in the broader fields of autism, epilepsy, and cancer can improve the understanding and, therefore, the treatment of TSC. This conference is important and timely in part because the TSC Clinical Consensus Conference held in June 2012 updated the recommendations for diagnosis, surveillance, and management of TSC. These recommendations reveal gaps in knowledge that need to be addressed by additional research. Based on previous, similar conferences, the 2013 research conference will attract >200 attendees, including approximately 20 invited speakers, 10 oral presentations selected from submitted abstracts, and 30 posters. The conference will bring together individuals who may otherwise not have an opportunity to meet. In addition to inviting speakers from outside the TSC field as noted above, from submitted abstracts the organizing committee will select 15 trainees or junior investigators to receive trave awards to offset transportation, hotel, and registration costs. On Saturday morning during the conference, the TS Alliance will host the second annual National Walk on the Mall in Washington, DC, providing a unique opportunity for researchers to engage directly with individuals affected by TSC. This conference maintains the structural and logistical features of previously successful conferences hosted by the TS Alliance while changing the topics and invited participants in important ways. To ensure that concepts presented differ greatly from previous conferences, 72% of invited speakers for 2013 were not speakers at the 2011 conference, and 44% did not attend the 2011 conference at all. To ensure new knowledge and ideas are presented, 22% of invited speakers have no or only one publication on "tuberous sclerosis" in PubMed. Most sessions include non-clinical and clinical presentations to encourage thinking and interaction along the continuum of translational science. There are no simultaneous platform sessions, so all attendees can hear and discuss all oral presentations. Breaks of 30 minutes are designed to encourage networking. During two mealtimes, panel discussions will enable interactive question-and-answer sessions with senior researchers from pharmaceutical and biotech companies of varying size on one day and with senior leaders of NCATS on another day. Working groups will discuss and develop actions to address key problems. Success of the 2013 conference will be measured by the impact of implementing recommendations of working groups and the acceleration of research through collaborative projects discussed during the conference.