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AA&MDSIF Second Bone Marrow Failure Disease Scientific Symposium

$27,000R13FY2009HLNIH

Aplastic Anemia & Mds International Fdn, Rockville MD

Investigators

Abstract

DESCRIPTION (provided by applicant): Aplastic anemia (AA), the myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that all result in bone marrow failure. When the marrow fails to produce blood cells, anemia, thrombocytopenia, and neutropenia can lead to debilitating symptoms and death. Once considered distinct, these three diseases are now believed to be linked by similar pathophysiologies. Exploration of current research issues in AA, MDS, and PNH would greatly benefit from increased collaboration between basic and clinical scientists, and between scientists studying the individual diseases. Increased understanding of the molecular events driving these diseases and of the response to treatment are needed to define at-risk populations and improve current therapies. The Aplastic Anemia &MDS International Foundation, Inc. (AA&MDSIF) proposes to hold its second Bone Marrow Failure Disease Scientific Symposium in the fall of 2009, with Richard M. Stone, M.D. from Dana-Farber Cancer Institute as Principal Investigator and Neal S. Young, M.D. from the National Heart, Lung, and Blood Institute as Co-Chair. The symposium will bring together physicians treating these diseases and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of controversy, share recent research results, and propose specific recommendations for the highest priority directions for basic and clinical research needed to advance the field. The format will consist of six presentation sessions, with time at the end of each session for questions and discussions, as well as for capture of recommendations for future research. The two days of the conference will also include poster and discussion sessions, and networking opportunities. The proceedings, including research recommendations, will be published online on the AA&MDSIF website and made freely available to the public. (End of Abstract)

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