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

描述（由申请人提供）： 第六届世界研讨会将于2010年2月10日至12日在美国佛罗里达州的迈阿密举行。今年是连续第二年由NINDS共同举办研讨会。该研究会议是一个多学科论坛，介绍了溶酶体疾病（LD）基础科学，转化研究和临床试验的最新信息。会议的主题“从转化到试验的转变”强调了主要目标，即评估将实验室研究纳入人类治疗的机制和障碍。其他目标建立在此基础上，并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，从而提高对这些疾病的生化，免疫，遗传和临床表现的认识; 3）确定和讨论诊断测试，筛查和治疗的最新发现; 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成分研究和/或治疗这些疾病的特定方面，因此他们很少接触到在其当前感兴趣领域之外的领域所做的工作。这次会议允许分享知识和所有LD的进步，并提供了一个讨论治疗结果的机会。世界研讨会的协同性质的证据是最近由NIH资助的多中心赠款，以响应罕见疾病临床研究联盟（RDCRC）RFA。RDCRC包括18个独特的项目，代表14个机构。除了临床医生和研究人员，世界研讨会欢迎患者，父母，护理人员和所有希望参加的患者倡导者。这种独特的功能允许一个小的，地理上分散的患者群体访问LD的最新科学进展的信息，并使科学家和临床医生的前所未有的访问临床环境以外的患者;提供一个论坛，否则不存在。2010年世界研讨会计划将分为五个部分：第一和第二部分，基础科学;第三和第四部分，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者会议，研究者的项目是RDCRC的一部分。每天六个私家侦探的将提出最新的进展情况，正在对他们的特定项目。此外，这些分组会议给每个研究人员一个与其他人分享想法的机会。今年的主题演讲将由计划委员会挑选的LD研究先驱发表。对于每一个会议，计划委员会已经选择了两个特邀演讲者，并将填补从提交摘要（摘要提交截止日期2009年10月1日）程序的其余部分。 公共卫生相关性：罕见孤儿疾病（如溶酶体疾病）的治疗相对较新，而且非常昂贵。一些终身治疗可能花费200，000 - 1，800，000美元每年为每个人。对于其他人来说，没有治疗方法;对于这种缓慢进展的身体和神经系统疾病，治疗方法的创新至关重要。世界研讨会将研究人员、医生、患者和患者倡导者聚集在一起，推动针对此类毁灭性疾病的医学科学和公共政策。

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.