13th International NCL Congress: Supporting US Based Scientists

描述（由申请人提供）：神经元样脂褐质病（NCLs， Batten病）是一组至少十种致命的遗传性疾病，主要影响儿童和年轻人。近年来，遗传缺陷已在至少8种形式的NCL中被确定，这导致了对这些疾病发病机制的理解和设计实验性治疗的进展。关于神经元样脂褐细胞病（巴顿病）的国际大会大约每两年举行一次，是唯一一个将那些对这组严重致残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NCL2012 （www.ncl2012.org）将于2012年3月在英国伦敦皇家霍洛威医院举行，但将采用一种新的独特形式，与第一次全球巴顿病家长组织会议同时举行。其目的是在最高水平上进行学术和临床研究的介绍和讨论，同时也为家长提供一个论坛，以方便的形式会面和获取最新信息。随着科学研究的步伐加快，我们离将这些方法转化为临床越来越近，迫切需要将这些进展清晰有效地传达给那些对其结果最感兴趣的人。NCL2012的演讲将由科学组织委员会从提交的摘要中选择。这将确保年轻的研究人员和NCL领域的新手将有同样的机会被选中，与那些在该领域建立良好的研究人员一样。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的联合会议上以一种易于理解的方式总结和介绍主要调查结果。这将与科学会议同时进行，由这些组织制定和领导一个项目，这些组织正在筹集资金，以支持来自世界各地的家庭出席会议。会议地点被选择为提供方便的校园住宿和现代化的会议设施，最大限度地提高了在主要国际和国内交通联系的地点进行互动的机会。与前几年一样，该申请是为了资助美国科学家参加NCL2012会议，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。

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.