The Alport Syndrome Symposium for Physicians, Researchers and Families

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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.