The Alport Syndrome Symposium for Physicians, Researchers and Families

描述(由申请人提供)摘要Alport综合征医师、研究人员和家庭研讨会(研讨会)将于2010年8月28日与国际儿科肾病学会(IPNA)第15届大会同时召开，时间为2010年8月29日至9月2日(纽约，纽约)。该研讨会是位于明尼苏达大学的Alport综合征治疗和结果登记处(ASTOR)和Alport综合征基金会(ASF)共同努力的成果。这项建议正在寻求资金，以：(1)将Alport综合征研究人员的国际小组与受影响的患者和家庭聚集在一起；(2)为临床科学家、基础研究人员、儿科肾病学家和家庭提供有关Alport综合征的遗传学、临床特征、发病机制、诊断和治疗的最新信息；(3)吸引初级研究人员和受训人员参与Alport综合征研究；(4)在患者和家庭、临床医生和研究人员之间建立合作，以促进有效招募临床试验人员；(5)通过与该领域许多最顶尖的临床医生、科学家和基础研究人员的互动，教育患者和家属并增强他们的能力。研讨会旨在激发人们对Alport综合征疗法临床试验的热情和支持，以及有效的招募战略。阿尔波特综合征是终末期肾脏疾病的重要遗传原因，目前还没有得到证实的治疗方法，尽管动物研究表明有几种潜在的有益干预措施，包括肾病学家常规使用的药物，如血管紧张素转换酶抑制剂。任何针对Alport综合征的干预试验都需要Alport患者及其家属以及他们的儿科肾病医生的积极参与和参与，以实现招募目标。对阿尔波特社区进行教育，使他们了解我们目前对这种疾病的了解，以及延缓终末期肾病的有效干预的前景，这对于为临床试验努力提供广泛支持至关重要。ASTOR和ASF选择第15届IPNA大会作为研讨会的场合，是因为它位于交通便利的主要人口中心，而且大会提供了吸引儿科肾病专家以及Alport患者和家庭的机会。研讨会将为招募足够数量的受影响个体进行临床试验所需的研究人员和家庭之间建立必要的国际合作提供一个论坛，将促进基础和临床研究产生的见解的分享，并为感兴趣的参与者提供一个讨论诊断和治疗问题并开始确定未来研究方向的机会。 公共卫生相关性：项目说明：公共卫生相关性尽管在阿尔波特综合征的动物模型中进行了大量研究，确定了各种潜在有益的干预措施，但目前还没有有效的治疗阿尔波特肾病的方法。面向医生、研究人员和家庭的Alport综合征研讨会将把医生和研究人员聚集在一起，分享有关Alport综合征的遗传学、临床特征、发病机制、诊断和治疗的最新信息，并吸引新的研究人员进入该领域。研讨会将提供一个平台，在研究人员和家庭之间建立必要的国际合作，以便在疾病的早期阶段招募足够数量的受影响个人，并将促进分享基础和临床研究产生的见解，以开始确定未来的研究方向。

DESCRIPTION (provided by applicant): ABSTRACT The Alport Syndrome Symposium for Physicians, Researchers, and Families (Symposium) will convene on August 28, 2010, in conjunction with the 15th Congress of the International Pediatric Nephrology Association (IPNA), August 29 - September 2, 2010 (New York, NY). The Symposium is a joint effort of the Alport Syndrome Treatments and Outcomes Registry (ASTOR), located at the University of Minnesota, and the Alport Syndrome Foundation (ASF). This proposal is seeking funding to: (1) bring together an international group of Alport syndrome researchers with affected patients and families; (2) provide clinician-scientists, basic researchers, pediatric nephrologists and families with the most recent information on the genetics, clinical features, pathogenesis, diagnosis and treatment of Alport syndrome; (3) attract junior investigators and trainees to Alport syndrome research; (4) build collaborations among patients and families, clinicians and researchers that will promote effective recruitment for clinical trials and (5) educate and empower patients and families through interaction with many of the foremost clinician-scientists and basic investigators in the field. The Symposium aims to generate enthusiasm and support for clinical trials of Alport syndrome therapies as well as effective recruitment strategies. Alport syndrome is an important genetic cause of end stage kidney disease for which there is no proven therapy, despite animal studies suggesting several potentially beneficial interventions, including medications used routinely by nephrologists such as angiotensin converting enzyme inhibitors. Any intervention trial in Alport syndrome will require the active engagement and participation of Alport patients and their families, as well as their pediatric nephrologists, in order to realize recruitment targets. Education of the Alport community regarding our current understanding of the disease and prospects for effective intervention to delay end stage kidney disease will be crucial for developing broad support for clinical trial efforts. ASTOR and the ASF selected the 15th IPNA Congress as the occasion for the Symposium because of its location in an easily accessible major population center and because of the opportunity provided by the Congress to attract pediatric nephrologists as well as Alport patients and families. The Symposium will provide a forum for establishing the necessary international collaboration among researchers and families required to recruit sufficient numbers of affected individuals for clinical trials, will promote the sharing of insights resulting from basic and clinical research, and offer an opportunity for interested constituents to discuss diagnosis and treatment issues and begin to identify future research directions. PUBLIC HEALTH RELEVANCE: Project Narrative: Public Health Relevance There is currently no effective treatment for Alport kidney disease in people, despite numerous studies in animal models of Alport syndrome that have identified a variety of potentially beneficial interventions. The Alport Syndrome Symposium for Physicians, Researchers, and Families will bring together physicians and researchers to share current information on the genetics, clinical features, pathogenesis, diagnosis and treatment of Alport syndrome and to attract new investigators to the field. The Symposium will provide a platform for establishing the necessary international collaboration among researchers and families required to recruit sufficient numbers of affected individuals at early stages of disease and will promote the sharing of insights resulting from basic and clinical research to begin to identify future research directions.