WORLD Symposium 2010 (Lysosomal Disease Network's 6th Annual Research Meeting)

简介（由申请人提供）：第六届年度世界研讨会将于2010年2月10-12日在美国佛罗里达州迈阿密举行。今年是该专题讨论会连续第二年由国家传染病研究所共同主办。本次研究会议是一个多学科论坛，介绍了溶酶体疾病（LD）的基础科学、转化研究和临床试验的最新信息。会议的主题是“从翻译到试验的过渡”，强调主要目的是评估将实验研究用于人类治疗的机制和障碍。在此基础上，进一步实现溶酶体疾病网络（LDN）的目标：1)进一步发展LDN的机制和使命；2)促进科学家之间的跨学科合作，提高对这些疾病的生化、免疫学、遗传学和临床表现的认识；3)识别和讨论诊断检测、筛查和治疗方面的最新发现；4)确定需要更多基础/临床研究、公共政策和监管关注的领域。由于许多LDN成员调查和/或治疗这些疾病的特定方面，他们很少接触到在其当前感兴趣的领域之外的领域所做的工作。这次会议为所有LD分享知识和进展提供了机会，并提供了讨论治疗结果的机会。世界研讨会协同性质的证据是最近由美国国立卫生研究院响应罕见疾病临床研究协会（RDCRC） RFA资助的多中心拨款。RDCRC包括18个独特的项目，代表14个机构。除临床医生和研究人员外，世界研讨会欢迎希望参加的患者、家长、护理人员和所有患者倡导者。这一独特的功能允许小的、地理上不同的患者群体获得有关LD最新科学进展的信息，并使科学家和临床医生能够前所未有地在诊所外接触患者；提供一个不存在的论坛。2010年世界专题讨论会计划分为五个部分：第一和第二部分，基础科学；第三和第四届会议：转化研究；第五和第六部分，临床试验。此外，每天下午的会议将包括研究人员会议，他们的项目是RDCRC的一部分。每天六个私家侦探的记者将介绍他们特定项目的最新进展。此外，这些分组会议给每个调查员一个与他人分享想法的机会。今年的主题演讲将由项目委员会选出的LD研究先驱发表。每一届会议，计划委员会将选出两名受邀演讲者，并从提交的摘要（摘要提交截止日期为2009年10月1日）中填补计划的剩余部分。

DESCRIPTION (provided by applicant): The 6th annual WORLD Symposium will be held February 10-12, 2010 in Miami, FL, USA. This year, for the second straight year, the Symposium is being co-organized by NINDS. This research meeting is a multidisciplinary forum presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases (LD). The theme of the meeting "Transitions from Translation to Trials" emphasizes the primary aim which is to assess the mechanisms, and obstacles, for taking bench research into human therapy. Additional aims build upon this, and implement the goals of the Lysosomal Disease Network (LDN) to: 1) Further develop the mechanisms and mission of the LDN; 2) Foster interdisciplinary collaboration between scientists, leading to improved knowledge regarding the biochemical, immunologic, genetic, and clinical manifestations of these diseases; 3) Identify and discuss the latest findings in diagnostic testing, screening, and treatment; 4) Identify areas that need more basic/clinical research, public policy, and regulatory attention. Because many LDN constituents investigate and/or treat specific aspects of these diseases they have little exposure to work done in areas outside their current area of interest. This meeting allows for the sharing of knowledge and advances for all LD, and provides an opportunity to discuss treatment outcomes. Proof of the synergistic nature of the WORLD Symposium is the multi-center grant recently funded by the NIH in response to the Rare Diseases Clinical Research Consortia (RDCRC) RFA. The RDCRC includes 18 unique projects, representing 14 institutions. In addition to clinicians and researchers the WORLD Symposium welcomes patients, parents, care-givers, and all patient advocates who wish to attend. This unique feature allows a small, geographically divergent patient population access to information on the latest scientific advances in LD and gives scientists and clinician's unprecedented access to patients outside the clinic setting; providing a forum that otherwise does not exist. The WORLD Symposium 2010 program will be organized in five sessions: Session I and II, Basic Science; Session III and IV, Translational Research; Session V and VI, Clinical Trials. Additionally, each afternoon session will include with a meeting of the Investigators who's projects are a part of the RDCRC. Each day, six P.I.'s will present updates on the progress being made on their particular project. Additionally, these breakout meetings give each investigator a chance to share ideas with the others. This year's Keynote Address will be delivered by a selected by the Program Committee, a pioneer in LD research. For each session, the Program Committee has selected two Invited Speakers and will fill the remainder of the program from submitted abstracts (abstract submission deadline October 01, 2009). PUBLIC HEALTH RELEVANCE: Treatments for rare, orphan diseases such as lysosomal diseases are relatively new and very expensive. Some life-long treatments can cost $200,000 - $1,800,000 per year for each individual. For others, there are no treatments; for such slowly progressive physical and neurologic disorders innovations in therapy are essential. The WORLD Symposium brings together researchers, physicians, patients and patient advocates to advance medical science and public policy for such devastating diseases.