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[Congressional Bills 110th Congress]
[From the U.S. Government Publishing Office]
[H. Res. 524 Engrossed in House (EH)]

                In the House of Representatives, U. S.,

                                                      October 15, 2007.
Whereas Diamond-Blackfan Anemia (``DBA'') is a rare genetic bone marrow failure 
        disorder affecting children and adults, 90 percent of whom are younger 
        than 1 year of age when they are diagnosed, and results in severe anemia 
        due to failure to produce red blood cells;
Whereas individuals and families suffering with rare diseases such as DBA not 
        only face the challenges of their debilitating and life-threatening 
        diseases, but must also confront the consequences of their rare disease 
        status;
Whereas individuals suffering from rare diseases need access to treatment 
        options and the potential for a cure;
Whereas research is proving the study of complex, rare diseases such as DBA 
        yield tremendous advancements in other, larger disease areas that affect 
        millions of Americans;
Whereas the children living with DBA have an increased risk of leukemia, solid 
        tumors, and complete bone marrow failure, and 50 percent of patients 
        with DBA are born with birth defects including abnormalities to the 
        face, head, upper arm and hand, genitourinary, and heart with 21 percent 
        of affected patients having more than 1 defect;
Whereas the study of DBA will yield the true incidence of aplastic anemia, 
        myelodysplastic syndrome, leukemia, and the predisposition to cancer in 
        DBA and will serve as an important model for understanding the genetics 
        of birth defects;
Whereas treatments for DBA, including the use of steroids (such as prednisone) 
        and blood transfusions, have potential long-term side effects, including 
        osteoporosis, impaired growth because of the steroids, diabetes, and 
        iron overload because of the transfusions;
Whereas the only cure for DBA is a bone marrow transplant, a procedure that 
        carries serious risks and, since most patients lack an acceptable donor, 
        is an option available for only about 25 percent of patients;
Whereas rare diseases, such as DBA, benefit greatly from well-established 
        comprehensive care centers such as the DBA Comprehensive Clinical Care 
        Center at Schneider Children's Hospital in New Hyde Park, New York (the 
        ``Center''), which has become the multidimensional hub for the care and 
        treatment of DBA patients across the country, as well as the home of the 
        DBA Patient Registry which has become a valuable national resource for 
        investigators utilizing the Center to accomplish research in a multitude 
        of areas not specific only to DBA;
Whereas the successful establishment of the Center became a model for how to 
        diagnose, treat, and improve the lives of patients with rare diseases, 
        while learning from the disorder to yield advancements in other areas of 
        disease research;
Whereas the success of the initial Center prompted the Centers for Disease 
        Control and Prevention's DBA Public Health Outreach and Surveillance 
        Program to establish 3 additional DBA Centers in Texas, California, and 
        Massachusetts to further patient access to information, treatment, and 
        care by DBA experts, which has resulted in a doubling of patient care 
        visits for DBA care and surveillance since their establishment;
Whereas the DBA Public Health Outreach and Surveillance Program at the Centers 
        for Disease Control and Prevention (``CDC'') has resulted in the 
        completion of the first CDC brochure for the DBA patient population, the 
        introduction of a DBA hotline and dedicated DBA nurse, and has resulted 
        in a 25-percent increase of enrollment of DBA patients into the DBA 
        Patient Registry in the first 2 years of the program;
Whereas the collaboration between the National Institutes of Health and the 
        Centers for Disease Control and Prevention and their close collaboration 
        with the Daniella Maria Arturi Foundation and the DBA Foundation have 
        driven the many recent successes in the DBA field and serve as a model 
        for addressing rare disease research efforts through close public and 
        private collaboration to achieve the highest levels of success in the 
        areas of improved patient care and disease research;
Whereas the interagency collaboration achieved within the National Institutes of 
        Health between the National Heart, Lung, and Blood Institute, the 
        National Institute of Diabetes and Digestive and Kidney Diseases, the 
        National Cancer Institute, and the Office of Rare Diseases to advance 
        the research and understanding of DBA has resulted in significant 
        advancements not only in the DBA scientific arena, but in understanding 
        its many links to more prevalent disorders; and
Whereas the DBA research initiatives have already yielded tremendous success 
        including the discovery of 2 ribosomal protein (``RP'') genes and the 
        identification that DBA is the first human disease linked to a ribosomal 
        protein problem which, as a fundamental unit of cellular function, has 
        been implicated in a wide range of human disorders including cancer, 
        making this discovery a profound example of the additional benefits that 
        may result from the study of DBA: Now, therefore, be it
    Resolved, That the House of Representatives--
            (1) recognizes that the identification of Diamond-Blackfan Anemia 
        (``DBA'') may advance the understanding of DBA, identify implications of 
        cancer predisposition, and serve as an important model for understanding 
        human development and the molecular basis for certain birth defects;
            (2) recognizes the importance of comprehensive care centers in 
        providing complete care and treatment for each patient, leading to an 
        increase in correct and early diagnosis;
            (3) commends Schneider Children's Hospital for providing the first 
        DBA Comprehensive Clinical Care Center for patients across the country, 
        for developing the DBA Patient Registry which has proven a robust 
        surveillance tool to understand the epidemiology, biology, and treatment 
        of DBA, and for proving a valuable resource for investigators at a 
        national level, working to understand DBA's link to more prevalent 
        disorders facing Americans;
            (4) commends the Daniella Maria Arturi Foundation and the Diamond-
        Blackfan Anemia Foundation for their efforts to facilitate the 
        successful collaboration among the National Institutes of Health and the 
        Centers for Disease Control and Prevention to achieve a successful 
        multidisciplinary approach between clinical and scientific DBA efforts 
        with the goal of shortening the life cycle of success realized between 
        the laboratory and applied patient care; and
            (5) encourages research efforts to further understand ribosomal 
        protein deficiencies in rare inherited diseases and to advance the 
        treatment options available to those with DBA.
            Attest:

                                                                          Clerk.
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