13th International NCL Congress: Supporting US Based Scientists

描述（由申请人提供）：神经元蜡样脂褐质病（NCL，Batten病）是一组至少10种致命的遗传性疾病，主要影响儿童和年轻人。近年来，已在至少8种形式的NCL中鉴定出遗传缺陷，这导致了对这些疾病的发病机制的理解和设计实验疗法的进展。神经元蜡样脂褐质沉积症（Batten病）国际大会大约每两年举行一次，是唯一一个将对这组严重致残性疾病的基础科学和临床护理感兴趣的人聚集在一起的论坛。下一次会议，NCL 2012（www.ncl2012.org），将于2012年3月在英国皇家霍洛威伦敦举行，但采用了一种新的和独特的形式，与第一次世界范围的巴滕病父组织会议一起举行。其目的是让学术和临床研究的最高水平的介绍和讨论，但也提供了一个论坛，家长见面，并在一个无障碍的格式访问最新的信息。随着科学研究的步伐加快，我们更接近将这些方法转化为临床，迫切需要将这些进展明确有效地传达给那些对其结果最感兴趣的人。NCL 2012的演讲将完全由科学组织委员会从提交的摘要中选出。这将确保年轻的研究者和那些新进入NCL领域的研究者将有与那些在该领域有良好声誉的研究者相同的机会被选中。会议将分为七个主要会议，每个会议将由每个领域的领导者主持和领导。这些主席还将在与家长组织会议举行的联席会议上以无障碍的方式总结和介绍主要调查结果。这将与科学会议同时举行，由这些组织制定和领导一个计划，筹集资金支持来自世界各地的家庭参加。会议地点的选择是为了提供无障碍的校园住宿和现代化的会议设施，最大限度地提高互动的机会，在一个网站，是在主要的国际和国家交通连接方便到达。重点是支持研究生和早期博士后科学家，而不是更成熟的研究人员。 公共卫生相关性：这次会议将首次汇集研究致命儿童疾病的研究人员，照顾这些儿童的临床医生以及这些儿童父母经营的非营利组织。这将提供一个独特的论坛，以促进这些群体之间的沟通，讨论研究和临床护理的最新进展，并提供对其影响的更好理解。

DESCRIPTION (provided by applicant): The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are a group of at least ten fatal inherited diseases that mostly affected children and young adults. In recent years the genetic defect has been identified in at least 8 forms of NCL and this has lead to advances in understanding the pathogenesis of these disorders and in devising experimental therapies. An International Congress on Neuronal Ceroid Lipofuscinosis (Batten disease) has been held approximately every two years and is the only forum brings together those with interests in basic science and clinical care for this group of profoundly disabling diseases. The next of these meetings, NCL2012 (www.ncl2012.org), will be held at the Royal Holloway London, UK in March 2012, but adopting a new and distinctive format by running together with the first worldwide meeting of Batten disease Parent Organizations. The aim is to allow presentation and discussion of academic and clinical research at the highest level, but also provide a forum for parents to meet and access the latest information in an accessible format. As the pace of scientific research accelerates and we move closer to translating these approaches into the clinic, there is a pressing need to communicate these advances clearly and effectively to those with the most vested interest in their outcome. The talks for presentation at NCL2012 will be selected by the scientific organizing committee entirely from submitted abstracts. This will ensure that young investigators and those new to the NCL field will have the same opportunities to be chosen to present as those investigators who are well established in the field. The conference will be split into seven main sessions each of which will be chaired and lead by key people who are leaders in each area. These chairs will also summarize and present the main findings in an accessible fashion in a joint session with the Parent's Organization Conference. This will run in parallel to the Scientific Conference, with a program set and led by these organizations, which are raising funds to support the attendance of families from around the world. The conference venue is chosen to provide accessible on-campus accommodation and modern conference facilities, maximizing the opportunities for interaction at a site that is within easy reach of major international and national transport links As in previous years, this application is made to fund US based scientists to attend the NCL2012 meeting, with an emphasis to be placed upon supporting graduate students and early postdoctoral scientists rather than more established investigators. PUBLIC HEALTH RELEVANCE: This conference will bring together for the first time researchers who study a fatal childhood disease, the clinicians who care for these children, and non-profit organizations run by these children's parents. This will provide a unique forum to promote communication between these groups, discuss the latest advances in research and clinical care, and provide a greater understanding of their implications.