House Passes McCarthy Legislation Supporting Diamond Blackfan Anemia Initiatives
Press Release
House Passes McCarthy Legislation Supporting Diamond Blackfan Anemia Initiatives
Earlier this week, the House unanimously agreed to legislation introduced by Rep. Carolyn McCarthy (H. Res. 524) supporting federal and private initiatives into Diamond Blackfan Anemia research and support.
Diamond Blackfan Anemia, or DBA, is a rare genetic bone marrow failure disorder that affects children and adults, stopping the body's ability to produce red blood cells. 90% of those suffering this disease were younger than a year old when they were diagnosed.
"Children living with DBA have an increased risk of leukemia, solid tumors, and complete bone marrow failure," said McCarthy. "The individuals and families suffering from rare diseases such as DBA not only face the challenges of their debilitating and life-threatening diseases, but must also confront the limited treatment and research options."
Researchers believe the study of DBA will not only unlock a cure, but will also yield clues to several other widespread diseases, providing valuable insights into the biology of blood disorders, blood cell formation, leukemia, and serve as an important model for understanding the genetics of birth defects.
"The only cure for DBA is a bone marrow transplant, a procedure that carries serious risks," said McCarthy. "Since most patients lack an acceptable donor, is an option available for only about 25% of patients."
Since rare diseases such as DBA do not receive the attention of more widespread ailments, research and treatment facilities are often limited. Fortunately, Long Island is home to the DBA Comprehensive Clinical Care Center at Schneider Children's Hospital in New Hyde Park, which has become the hub for the care and treatment of DBA patients across the country. The facility is also home of the DBA Patient Registry, which has become a valuable national resource for families and researchers.
"The successes made at Schneider's Children's Hospital have prompted the Centers for Disease Control and Prevention's DBA Public Health Outreach and Surveillance Program to establish three additional DBA Centers throughout the country," said McCarthy. "This has resulted in a doubling of patient care visits for DBA since their establishment."
H. Res. 524 expresses Congress' support for DBA research and initiatives, and provides federal momentum for a cure. According to McCarthy, "With passage of this legislation, we in Congress have given all families suffering from this rare disease a chance at hope."
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