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

2010 年世界研讨会（溶酶体疾病网络第六届年度研究会议）

• 批准号: 7915961
• 负责人: Chester B. Whitley
• 金额: $ 3.5万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-02-09 至 2011-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7915961
• 关键词: Access to Information; Address; Advocate; Area; Attention; Basic Science; Biochemical; Caregivers; Clinic; Clinical; Clinical Research; Clinical Trials; Diagnostic tests; Disease; Exposure to; Fostering; Funding; Genetic; Goals; Grant; Human; Immunologics; Individual; Institution; Knowledge; Life; Medical; Mission; National Institute of Neurological Disorders and Stroke; Nature; Orphan Disease; Parents; Patients; Physicians; Public Policy; Rare Diseases; Research; Research Personnel; Science; Scientific Advances and Accomplishments; Scientist; Screening procedure; Translational Research; Translations; Treatment outcome; United States National Institutes of Health; Update; Work; abstracting; cost; improved; innovation; interdisciplinary collaboration; interest; meetings; multidisciplinary; nervous system disorder; patient population; programs; public health relevance; response; symposium

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.

描述（由申请人提供）：第六届年度世界研讨会将于2010年2月10日至12日在美国佛罗里达州迈阿密举行。今年，连续第二年，研讨会由Ninds共同组织。这次研究会议是一个多学科论坛，介绍了基础科学，转化研究和溶酶体疾病临床试验（LD）的最新信息。会议“从翻译到试验的过渡”的主题强调，主要目的是评估对人类治疗进行替补研究的机制和障碍。其他目标是建立在此基础上，并实施溶酶体疾病网络（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，从而提高了有关这些疾病的生化，免疫，遗传和临床表现的知识； 3）识别并讨论诊断测试，筛查和治疗中的最新发现； 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成分调查和/或治疗这些疾病的特定方面，因此在目前感兴趣的领域以外的领域所做的工作很少。这次会议允许分享所有LD的知识和进步，并提供了讨论治疗结果的机会。世界研讨会的协同性证明是NIH最近为罕见疾病临床研究联盟（RDCRC）RFA资助的多中心赠款。 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，世界研讨会还欢迎患者，父母，照顾者以及所有希望参加​​的患者拥护者。这一独特的功能允许患者群体访问有关LD最新科学进步的信息，并为科学家和临床医生提供了前所未有的访问诊所环境中的患者的信息；提供否则不存在的论坛。 2010年世界研讨会计划将在五个课程中组织：第I和II会，基础科学；第三会议和IV，翻译研究； V和VI会议，临床试验。此外，每个下午的会议将包括调查人员的会议，该项目的项目是RDCRC的一部分。每天，六个P.I.都会介绍其特定项目的进度。此外，这些突破性会议使每个调查员有机会与他人分享想法。今年的主题演讲将由计划委员会选定的LD研究先驱计划委员会发表。对于每个会议，计划委员会都选择了两名受邀发言人，并将从提交的摘要中填写该计划的其余部分（摘要提交截止日期，2009年10月1日）。 公共卫生相关性：治疗罕见的孤儿疾病（例如溶酶体疾病）相对较新且非常昂贵。每个人的终身治疗费用为$ 200,000- $ 1,800,000。对于其他人来说，没有治疗；对于这种缓慢进行性的身体和神经系统疾病，治疗中的创新至关重要。世界研讨会汇集了研究人员，医师，患者和患者倡导者，以推动医学和公共政策的这种毁灭性疾病。