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）：第九届年度世界研讨会将于2013年2月13日至15日（美国佛罗里达州奥兰多）举行。本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。本次会议允许分享所有溶酶体疾病的知识和进展，并提供了讨论治疗结果的机会。世界研讨会的协同性质的证据是多中心联盟“溶酶体疾病网络”（U 54 NS 065768，罕见疾病临床研究网络RDCRN的成员）的产物。除了研究人员和临床医生，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获得有关溶酶体疾病的最新科学进展的信息，并使科学家和临床医生前所未有地接触到临床环境之外的患者;提供一个论坛来分享信息，否则不存在。该计划分为六个阶段，从“基础研究”到“转化研究”再到“临床研究”。对于每一个，计划委员会将选择两个特邀演讲者，并将填补从提交摘要（提交截止日期2012年10月1日）的程序的其余部分。连续第二年，研讨会将在研究会议之前的星期二（2月12日）增加一个教学部分“溶酶体101，溶酶体生物学和疾病的基础”。在前一年的课程基础上，但不是取代，这个4小时的教育会议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，为随后的前沿研究提供共同的理解。为了促进资深研究者和年轻研究者之间的关系，我们将再次赞助年轻研究者的旅行，以促进他们参加“Lysosomes 101”和世界会议;指导新科学家是LDN和RDCRN的主要目标。