2013 International Research Conference on Tuberous Sclerosis Complex and Related

2013年结节性硬化症及相关疾病国际研究会议

• 批准号: 8529364
• 负责人: Steven L Roberds
• 金额: $ 2.55万
• 依托单位: TUBEROUS SCLEROSIS ALLIANCE
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-04-15 至 2014-04-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8529364
• 关键词: Acceleration; Address; Affect; Autistic Disorder; Award; Basic Science; Brain Neoplasms; Clinical; Clinical Research; Collaborations; Communication; Consensus; Data; Diagnosis; Disease; District of Columbia; Ensure; Environment; Epilepsy; FDA approved; Faculty; Funding; Genetic; Genotype; Goals; Hearing; Individual; Industry; International; Kidney; Kidney Neoplasms; Knowledge; Learning; Lung; Lymphangioleiomyomatosis; Malignant Neoplasms; Measures; Medical; Methods; Neurologic; Oral; Organ; Outcome; Outcome Measure; Participant; Pharmaceutical Preparations; Pharmacologic Substance; Phenotype; PubMed; Publications; Qualifying; Quality of life; Rare Diseases; Recommendation; Research; Research Personnel; SDZ RAD; Skin; Suggestion; Symptoms; Thinking; Translational Research; Transportation; Tuberous Sclerosis; Tuberous sclerosis protein complex; Update; Walking; Work; abstracting; base; cost; cytotoxic; data sharing; design; drug discovery; expectation; experience; health care quality; improved; insight; interest; mTOR Inhibitor; mTOR inhibition; meetings; neuropathology; posters; public health relevance; success; symposium; tumor; tumor growth; working group

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.

描述(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、淋巴管肌瘤病(LAM)、癌症和表型重叠的罕见疾病)患者未得到满足的医疗需求。由于与TSC相关的症状多种多样，对TSC的研究将增加与没有TSC的个体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，从而提高对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对TSC的诊断、监测和管理的建议。这些建议揭示了需要通过更多研究解决的知识差距。在以往类似会议的基础上，2013年的研究会议将吸引200人参加，其中包括大约20名受邀演讲者，从提交的摘要中挑选出10个口头报告，以及30张海报。这次会议将把原本可能没有机会见面的人聚集在一起。除了如上所述邀请TSC领域以外的演讲者外，组委会还将从提交的摘要中挑选15名学员或初级调查人员获得Trave奖，以抵消交通费、住宿费和注册费。在周六上午的会议期间，TS联盟将在华盛顿特区的购物中心举办第二届年度全国步行活动，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。本次会议保留了TS联盟以往成功举办的会议的结构和后勤特点，同时在重要方面改变了主题和受邀与会者。为了确保提出的概念与以往的会议有很大不同，2013年受邀发言者中有72%没有在2011年会议上发言，44%根本没有出席2011年会议。为了确保展示新的知识和想法，22%的受邀演讲者没有或只在PubMed上发表了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报告，以鼓励思维和互动沿翻译科学的连续体。没有同步的平台会议，因此所有与会者都可以听取和讨论所有口头演示。30分钟的休息时间是为了鼓励交流。在两次用餐时间，小组讨论将允许一天与来自不同规模的制药和生物技术公司的高级研究人员进行互动问答，另一天与NCATS的高级领导人进行互动问答。工作组将讨论和制定解决关键问题的行动。2013年会议的成功将以落实工作组建议的影响和通过会议期间讨论的合作项目加快研究的速度来衡量。