6th International Symposium on FAP Disorders and 5th...

描述(由申请人提供)： 我们请求支持将于2005年8月24日至26日在加利福尼亚州拉霍亚的斯克里普斯研究所举行的第六届家族性淀粉样多发性神经病和心肌病及其他转运蛋白相关疾病国际研讨会和第五届家族性淀粉样多发性神经病肝移植国际研讨会。 与过去一样，受邀者和参与者将包括临床医生、临床和研究调查人员以及来自许多国家和地区的患者群体的代表，以会面并讨论这些疾病的临床特征、诊断和管理。这次三年一度的会议是外科医生、非外科医生和研究人员会面并分享见解并讨论这些患者的治疗进展的唯一会议。会议的目标是： 1)进一步发展一些国际合作互动以及在过去的会议上发起的个人研究人员之间的更有限的互动，包括成立家族性淀粉样多发性神经病肝移植登记处，这是国际医学界相对独特的实体； 2)提出和讨论与新疗法开发有关的发病机制的新数据和新观点； 3)讨论最近启动的转甲状腺素纤维生成抑制剂体外国际试验的进展和未预料到的问题； 4)提供新的数据，以继续界定所有FAP共有的特征和特定前体独有的特征； 5)提供一个可以组织潜在治疗药物研究的环境，最大限度地增加可用的患者数量，并允许以标准格式分析在非同期试验中获得的结果。

DESCRIPTION (provided by applicant): We are requesting support for the combined Sixth International Workshop on The Familial Amyloidotic Polyneuropathies and Cardiomyopathies and Other Transthyretin Related Disorders and the Fifth International Workshop of Liver Transplantation in Familial Amyloid Polyneuropathy, to be held from August 24-26, 2005 at The Scripps Research Institute in La Jolla, California. As in the past, invitees and participants will include clinicians, clinical and research investigators, and representatives from patient groups from many countries to meet and discuss the clinical features, diagnosis, and management of these disorders. This triennial meeting is the only meeting where surgeons, non-surgeon physicians, and researchers meet and share insights and discuss progress in the treatment of these patients. Goals of the meeting are to: 1) Further develop a number of international cooperative interactions as well as more restricted interactions between and among individual investigators initiated at past meetings, including the formation of the Familial Amyloidotic Polyneuropathy Liver Transplantation Registry, a relatively unique entity in international medicine; 2) Present and discuss new data and ideas concerning pathogenesis that are related to development of new therapies; 3) Discuss accrual, progress and unanticipated problems of a recently initiated international trial of an in vitro inhibitor of transthyretin fibrillogenesis; 4) Present new data to continue to define those features common to all FAPs and those that are unique to specific precursors; 5) Provide a setting in which studies of potential therapeutic agents can be organized, maximizing the numbers of available patients and allowing the results obtained in non-contemporaneous trials to be analyzed in a standard format.