Lysosomal Disease Network-4th Annual WORLD Symposium

溶酶体疾病网络-第四届年度世界研讨会

• 批准号: 7483469
• 负责人: Chester B. Whitley
• 金额: $ 3万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-02-20 至 2009-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7483469
• 关键词: Advocate; Area; Attention; Basic Science; Clinical; Clinical Research; Clinical Sciences; Clinical Trials; Committee Members; Crying; Diagnostic tests; Disease; Ensure; Exposure to; Fostering; Functional disorder; Future; Geographic Locations; Goals; Guidelines; Human Resources; Individual; Innovative Therapy; Investigational Therapies; Knowledge; Life; Medical; Molecular Biology; Natural History; Orphan; Parents; Pathology; Patients; Physicians; Population; Public Health; Public Policy; Quality of life; Recommendation; Recruitment Activity; Research; Research Personnel; Science; Screening procedure; Services; Testing; Therapeutic; Translational Research; Treatment Cost; Treatment outcome; United States National Institutes of Health; Work; abstracting; day; diagnosis evaluation; improved; innovation; interdisciplinary collaboration; interest; member; multidisciplinary; nervous system disorder; programs; symposium

DESCRIPTION (provided by applicant): The 4th annual WORLD Symposium will be held February 13-15, 2008 in Las Vegas, NV, USA. In addition to the annual meeting of the Lysosomal Disease Network (LDN), the symposium will provide a multidisciplinary forum to present and discuss clinical research to foster new understanding of the molecular biology, pathology, and potential for treatment for these diseases. Specific aims are to: 1) Further coalesce members of the LDN as a functional research network, 2) Foster interdisciplinary collaboration with the overall goal of improving knowledge of basic discoveries and clinical manifestations of these diseases; 3) Identify and discuss the latest findings in the natural history of lysosomal diseases (LDs), diagnostic testing and screening, and treatment; and 4) Identify areas requiring additional basic/clinical research, public policy and regulatory attention. Many of the network's constituents investigate and/or treat specific aspects of these diseases but have little exposure to work done in areas outside their current area of interest. This meeting platform promotes the sharing of basic and clinical science advances for all LDs, and provides an opportunity to discuss the related treatment outcome issues. The study and treatment of LDs pose unique challenges due to small populations of patients being dispersed over a wide geographic area. Providing a forum to share information in the basic and clinical sciences will advance research in both realms, and result in a more effective and efficient dissemination of knowledge, clinical guidelines, and recommendations for diagnosis, evaluation, and treatment. The 2008 WORLD Symposium program will be organized in five sessions. Session I: Basic Science, Advances in Understanding the Pathophysiology of Lysosomal Diseases. Session II: Basic Science, Future Therapeutic Approaches to Treatment of Lysosomal Diseases. Session III: Translational Research, Innovative Therapies Moving Toward Clinical Trials for Lysosomal Diseases. Session IV: Clinical Trials, Experimental Therapies Currently Being Tested. Session V: Natural History, Treatment Outcomes, and Quality of Life Issues. Additional concurrent disease-specific sessions on the afternoon of the last day (of special interest to patients, parents, and patient advocates) will include educational presentations, support services, and round table discussions. To help ensure a high-quality scientific content, and fulfill the R13 requirement for NIH involvement, Dr. Danilo Tagle (NINDS) has agreed to participate on the Program Committee. Additional, intramural and administrative NIH personnel are being recruited to supplement LDN Steering Committee members who thus constitute the Program Committee. For each session, the Program Committee will select two Invited Speakers and fill the remainder of the program from submitted abstracts (abstract submission deadline June 1, 2008; program finalized July 1, 2008). PUBLIC HEALTH RELEVANCE: New, expensive treatments are developed for the rare, "orphan" lysosomal diseases, some life-long treatments costing $200,000 - $1,800,000 per year for each individual. For others, there are no treatments; such slowly progressive physical and neurologic disorders cry out for innovations in therapy. The WORLD Symposium brings together researchers, physicians and patient advocates to advance medical science and public policy for such desperate problems.

描述（由申请人提供）：第四届年度世界研讨会将于 2008 年 2 月 13 日至 15 日在美国内华达州拉斯维加斯举行。除了溶酶体疾病网络 (LDN) 年会外，研讨会还将提供一个多学科论坛来展示和讨论临床研究，以促进对分子生物学、病理学和这些疾病治疗潜力的新认识。具体目标是：1) 进一步将 LDN 成员合并为一个功能性研究网络，2) 促进跨学科合作，总体目标是提高对这些疾病的基本发现和临床表现的了解； 3) 识别并讨论溶酶体疾病（LD）自然史、诊断测试和筛查以及治疗方面的最新发现； 4) 确定需要额外基础/临床研究、公共政策和监管关注的领域。该网络的许多成员调查和/或治疗这些疾病的特定方面，但很少接触到他们当前感兴趣的领域之外的领域所做的工作。该会议平台促进所有LD分享基础和临床科学进展，并提供讨论相关治疗结果问题的机会。由于少数患者分布在广泛的地理区域，LD 的研究和治疗提出了独特的挑战。提供一个共享基础科学和临床科学信息的论坛将推进这两个领域的研究，并导致更有效和高效地传播知识、临床指南以及诊断、评估和治疗建议。 2008年世界研讨会计划将分为五场。第一节：基础科学，溶酶体疾病病理生理学的进展。第二节：基础科学，溶酶体疾病的未来治疗方法。第三场会议：转化研究、创新疗法走向溶酶体疾病临床试验。第四节：临床试验，目前正在测试的实验疗法。第五节：自然史、治疗结果和生活质量问题。最后一天下午举行的其他同期疾病特定会议（患者、家长和患者倡导者特别感兴趣）将包括教育演讲、支持服务和圆桌讨论。为了帮助确保高质量的科学内容，并满足 NIH 参与的 R13 要求，Danilo Tagle 博士 (NINDS) 已同意加入计划委员会。正在招募额外的 NIH 校内和行政人员来补充 LDN 指导委员会成员，从而组成计划委员会。对于每届会议，计划委员会将选出两名特邀演讲者，并从提交的摘要中填写计划的其余部分（摘要提交截止日期为 2008 年 6 月 1 日；计划最终确定日期为 2008 年 7 月 1 日）。公共健康相关性：针对罕见的“孤儿”溶酶体疾病开发了新的、昂贵的治疗方法，一些终身治疗的费用为每人每年 200,000 至 1,800,000 美元。对于其他人来说，没有治疗方法；这种缓慢进展的身体和神经系统疾病迫切需要治疗创新。世界研讨会汇集了研究人员、医生和患者权益倡导者，以推进医学科学和公共政策来解决这些紧迫的问题。