Diamond Blackfan Anemia International Consensus Conference

描述（由申请人提供）：

DESCRIPTION (provided by applicant): The Diamond Blackfan Anemia International Consensus Conference (DBA-ICC) is the only annual forum which brings together physicians, scientists, and other parties interested in all aspects of the congenital bone marrow syndrome Diamond Blackfan Anemia (DBA). DBA is the first human disease linked to a failure of ribosome synthesis. Understanding the pathophysiology of this rare disease has required an interdisciplinary approach bringing together the highly specialized fields composed of hematologists interested in congenital bone marrow failure syndromes and basic scientists interested in ribosome biology. The annual DBA-ICC meetings have fostered an environment that has led to numerous collaborations between these diverse groups providing a rich breeding ground for translational research. Over the past 10 years these conferences have helped create and disseminate advancements in patient care to the wider community of physicians who may only rarely encounter DBA in clinical practice. Similarly, research into the underlying molecular basis for DBA has led to a much greater understanding of the role of the ribosome in human disease, which has implications for other diseases including cancer. The broadening scope of DBA research has brought new investigators into the DBA field with the result that the DBA-ICC meetings continue to grow in popularity and relevance to physicians and scientists alike. As the annual meeting grows the organizers feel that new sources of funding are needed to maintain the standard of excellence established at past meetings. This proposal therefore represents a request for partial support of the upcoming DBA- ICC meeting from the National Institutes of Health through the R13 award mechanism. PUBLIC HEALTH RELEVANCE: The Diamond Blackfan Anemia International Consensus Conference is the only meeting held each year that brings together physicians, scientists, and other parties interested in all aspects of Diamond Blackfan anemia. Over the past 10 years this meeting has produced a consensus clinical care document for patient care, helped coordinate clinical trials, stimulated collaborations promoting translational research and fostered an environment which has led to tremendous advances in understanding the underlying molecular basis for Diamond Blackfan anemia. As Diamond Blackfan anemia is the first human disease caused by a failure of ribosome synthesis, an interdisciplinary conference such as this is absolutely essential for bringing together physicians and scientists of diverse disciplines for productive interactions relevant to DBA and broadening applications to other disease states.