Diamond Blackfan Anemia (DBA) is an extremely rare inherited bone marrow failure syndrome that affects less than 1000 people in the United States. This inherited condition is characterized by the inability of the bone marrow to produce a sufficient quantity of functioning red blood cells While some patients respond to steroid treatment, the majority of patients require monthly red blood cell transfusions to stay alive. The Diamond Blackfan Anemia Foundation, Inc. (DBAF) is a 501(c)(3) not-for-profit organization that was created in 1994 to raise funds for DBA research and education. The DBAF is a family operated organization with virtually all the funds raised by families and friends of those affected by DBA. The DBAF is requesting funding from the NIH National Institute of Diabetes and Digestive and Kidney Diseases for partial financial support for the 14th Diamond Blackfan Anemia International Consensus Conference (DBA-ICC) to be held at the Ritz-Carlton, Atlanta, GA on March 5 to 7, 2016. Specifically, $25,000 in funding is requested to provide support for the estimated 20 young investigators who will attend the conference. The DBA-ICC is a bi- annual conference that brings together clinicians and scientists from around the world to present and discuss the latest research on all aspects of Diamond Blackfan anemia (DBA). This conference also includes parents of children affected by DBA and on occasion, patients themselves. This mix of attendees provides a forum for the dissemination of state-of-the-art clinical care and cutting edge research to families affected by DBA. While the overall program for the 14th DBA-ICC is still under development, the conference will be organized to meet the following main objectives: ? Provide a venue for the exchange of ideas and foster new collaborations among DBA clinicians and scientists, ? Document results from ongoing clinical trials and coordinate efforts for new clinical trials, ? Bridge the gulf between clinicians and scientists working on DBA to promote translational research, ? Encourage young clinicians and scientists to enter into DBA as independent investigators, ? Expose clinicians and scientists to mechanisms of funding for DBA-related research. It is anticipated that the conference will include approximately 100 attendees (including 20 young investigators) from as many as 10 different countries. The technical program will be organized around the following themes and include a poster session for young investigators: ? Emerging Therapies in DBA, ? Model Systems of DBA, ? Pathogenic Mechanisms of DBA, ? Pathogenic Mechanisms and the Ribosome, ? The Role of GATA1 and DBA, ? Gene Discovery, ? Clinical Aspects of DBA, and ? Updates from the NHLBI & NIDDK.
Diamond Blackfan Anemia (DBA) is an extremely rare inherited bone marrow failure syndrome that affects less than 1000 people in the United States. DBA is characterized by the inability of the bone marrow to produce a sufficient quantity of functioning red blood cells. While some patients respond to steroid treatment, the majority of patients require monthly red blood cell transfusions. The bi-annual Diamond Blackfan Anemia International Consensus Conference provides a unique forum of interaction for the international community of researchers and clinicians who are committed to finding a cure and providing the best possible patient care for DBA. A major objective of the conference is to provide opportunities for young researchers to engage with leading scientists and clinicians. Because DBA is caused by mutations of ribosomal protein genes and because DBA patients have a much greater risk of developing cancer, the study of DBA is highly relevant to other fields of medical research.