14th International NCL Congress: Supporting US Based Scientists

描述(申请人提供)：神经性蜡样脂褐素增多症(NCLS，巴顿病)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，在至少8种形式的NCL中发现了这种基因缺陷，这导致了对这些疾病的发病机制的理解和实验治疗的设计方面的进展。神经性干酪样脂褐质沉着症(巴顿病)国际大会大约每两年举行一次，是聚集了对这一组严重致残性疾病的基础科学和临床护理感兴趣的人的唯一论坛。下一次会议NCL2014(http://nclcordoba.com/website/)，)将于2014年10月22日至26日在阿根廷科尔多瓦的科尔多瓦喜来登酒店举行。这也吸引了来自世界高、中、低收入国家的巴顿病母组织。我们的目标是提供一次将保持最高标准的科学和 临床演示，但同时以世界各地的NCL家庭、神经学家、儿科医生和护理人员可访问的格式呈现最佳临床标准的信息。这是第一次在美洲中等收入国家举行的大会，将科学知识和社会效益扩展到一个新的地区。科学委员会确定了主题的优先次序，并邀请了在这一领域做出重大贡献的非巴顿病领域的演讲者和该领域的专家，他们将就他们在这一领域的最新研究发表讲话。此外，初级研究人员和学生演讲将从提交的摘要中挑选出来，以确保年轻的研究人员和那些新进入NCL领域的人有机会出席这次国际会议。会议分为六个主要会议和两个研讨会，每个会议将由在每个领域担任领导人的关键人物共同主持和领导。这些主持人将在与家长组织会议的联席会议上以通俗易懂的方式总结和介绍主要调查结果。家长组织会议将与科学会议同时举行，并由这些组织制定和领导一个计划，这些组织正在筹集资金，以支持来自世界各地的家庭参加会议。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，最大限度地增加在距离主要国际和国家交通运输线路很容易到达的地点进行互动的机会。另外， 在会议期间，将向家庭提供训练有素的工作人员的支持和照顾。与往年一样，这项申请是为了资助美国科学家参加NCL2014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 eight forms of NCL and this has led to advances in understanding the pathogenesis of these disorders and in devising experimental therapies. An International Congress on Neuronal Ceroid Lipofuscinosis (Batten disease) is 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, NCL2014 (http://nclcordoba.com/website/), will be held at the Sheraton Cordoba Hotel in Cordoba, Argentina, from October 22-26, 2014. This also appeals to Batten Disease Parent Organizations from high, middle and low income countries of the world. Our goal is to provide a meeting that will retain the highest standard of scientific and clinical presentations, but to present at the same time information of the best clinical standards in a format that is accessible to NCL families, neurologists, pediatricians and caregivers around the world. It is first Congress to be held in a middle income country of the Americas, expanding the scientific knowledge and the social benefits to a new region. The scientific committee has prioritized topics and has invited both speakers not working on Batten disease that make major contributions in this area and experts in field whom will speak on their most recently published research in this area. Additionally, junior investigator and student talks will be selected from submitted abstracts, ensuring that young investigators and those new to the NCL field have an opportunity to present at this international conference. The conference is split into six main sessions and two workshops, each of which will be co-moderated and lead by key people who are leaders in each area. These moderators will summarize and present the main findings in an accessible fashion in a joint session with the Parent's Organization Conference. The Parent's Organization Conference 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. Additionally, support and care by trained staff will be made available to families during the conference. As in previous years, this application is made to fund US based scientists to attend the NCL2014 meeting, with an emphasis to be placed upon supporting graduate students and early postdoctoral scientists rather than more established investigators.