Lysosomal Disease Network-9th Annual WORLD Symposium

溶酶体疾病网络-第九届世界研讨会

• 批准号: 8456842
• 负责人: Chester B. Whitley
• 金额: $ 2.83万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-09-28 至 2013-09-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8456842
• 关键词: Access to Information; Achievement; Advocate; Area; Attention; Basic Science; Biochemical; Biochemistry; Biological Markers; Biology; Bone Diseases; Caregivers; Clinic; Clinical; Clinical Research; Clinical Trials; Collaborations; Development; Diagnostic tests; Disease; Economic Factors; Education; Educational Curriculum; Ethics; Evaluation; Event; Exposure to; Fostering; Funding; Genetic; Goals; Hour; Human; Image; Immunologics; Immunology; Individual; Knowledge; Life; Lysosomes; Medical; Mentors; Metabolic; Metabolism; Mission; Molecular Biology; Molecular Genetics; National Institute of Neurological Disorders and Stroke; Natural History; Nature; Neonatal Screening; Orphan Disease; Parents; Patients; Physicians; Principal Investigator; Progress Reports; Public Policy; Publishing; Rare Diseases; Research; Research Personnel; Research Project Grants; Schools; Science Policy; Scientific Advances and Accomplishments; Scientist; Screening procedure; Translational Research; Travel; Treatment outcome; United States National Institutes of Health; Work; abstracting; base; cost; improved; innovation; interdisciplinary collaboration; interest; lectures; meetings; member; multidisciplinary; nervous system disorder; patient population; programs; response; success; symposium

DESCRIPTION (provided by applicant): The 9th annual WORLD Symposium will be held February 13-15, 2013 (Orlando, FL, USA). This research meeting is a multidisciplinary forum presenting the latest advances in basic science, translational research, and clinical trials for lysosomal diseases. The theme of the meeting will emphasize 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 across all lysosomal diseases, and provides an opportunity to discuss treatment outcomes. Evidence of the synergistic nature of the WORLD Symposium has been outgrowth of the multi-center consortium "Lysosomal Disease Network" (U54 NS065768, a member of the Rare Diseases Clinical Research Network, RDCRN). In addition to researchers and clinicians, the WORLD Symposium welcomes patients, parents, caregivers, 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 lysosomal diseases and gives scientists and clinician's unprecedented access to patients outside the clinic setting; providing a forum to share information that otherwise does not exist. The program is organized in six sessions extending from "basic research" through "translational research" to "clinical research". For each, the Program Committee will select two Invited Speakers and will fill the remainder of the program from submitted abstracts (submission deadline October 1, 2012). For the second straight year, the Symposium will add a didactic component "Lysosomes 101, Fundamentals of Lysosomal Biology and Disease" on the Tuesday (February 12) preceding the research meeting. Building on-but not supplanting-the curriculum of the previous year, this 4-hour educational session will offer attendees the basic concepts of biochemistry, immunology, molecular biology, and genetics of lysosomal disease, providing a common understanding for subsequent cutting edge research presentations. In order to foster relationships between senior and young investigators, we will once again sponsor travel for young investigators facilitating their attendance of "Lysosomes 101" and the WORLD conference; the mentoring of new scientists is a major goal of the LDN and the RDCRN.

描述（由申请人提供）：第9届年度世界研讨会将于2013年2月13日至15日举行（美国佛罗里达州奥兰多）。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的最新进展。会议的主题将强调主要目的，即评估对人类治疗的基准研究的机制和障碍。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。这次会议允许共享所有溶酶体疾病的知识和进步，并提供了讨论治疗结果的机会。多中心财团“溶酶体疾病网络”（U54 NS065768，稀有疾病临床研究网络的成员，RDCRN）的成员，世界研讨会的协同性质的证据已经存在。除研究人员和临床医生外，世界研讨会还欢迎希望参加的患者，父母，看护人和所有希望参加​​的患者拥护者。这一独特的功能允许患者人群访问有关溶酶体疾病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诊断患者的机会；提供一个论坛来共享其他不存在的信息。该计划在六个课程中组织，从“基础研究”延伸到“转化研究”到“临床研究”。每个计划委员会将选择两名受邀发言人，并将从提交的摘要中填写该计划的其余部分（提交截止日期，2012年10月1日）。在研究会议之前，该研讨会连续第二年将增加一个教学成分“溶酶体101，溶酶体生物学和疾病的基本原理”。这次4小时的教育会议建立在上一年的课程中，但并未取代课程，将为与会者提供生物化学，免疫学，分子生物学和溶酶体疾病遗传学的基本概念，为随后的尖端研究提供了共同的理解。为了促进高级和年轻调查员之间的关系，我们将再次赞助年轻调查员旅行，以促进他们参加“溶酶体101”和世界会议的出席；新科学家的指导是LDN和RDCRN的主要目标。