H. Res. 524 
In the House of Representatives, U. S., 
October 15, 2007. 
Whereas Diamond-Blackfan Anemia (‘‘DBA’’) is a rare ge-
netic 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 ane-
mia 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 debili-
tating and life-threatening diseases, but must also con-
front 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 dis-
eases such as DBA yield tremendous advancements in 
other, larger disease areas that affect millions of Ameri-
cans; 
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 de-
fect;
•HRES 524 EH 
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 poten-
tial long-term side effects, including osteoporosis, im-
paired 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 pa-
tients 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 Reg-
istry 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 Cen-
ters for Disease Control and Prevention’s DBA Public
•HRES 524 EH 
Health Outreach and Surveillance Program to establish 3 
additional DBA Centers in Texas, California, and Massa-
chusetts to further patient access to information, treat-
ment, and care by DBA experts, which has resulted in 
a doubling of patient care visits for DBA care and sur-
veillance since their establishment; 
Whereas the DBA Public Health Outreach and Surveillance 
Program at the Centers for Disease Control and Preven-
tion (‘‘CDC’’) has resulted in the completion of the first 
CDC brochure for the DBA patient population, the intro-
duction 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 Preven-
tion 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 pa-
tient 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 Na-
tional 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
•HRES 524 EH 
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 dis-
covery 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 under-
standing of DBA, identify implications of cancer pre-
disposition, and serve as an important model for under-
standing 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 re-
source for investigators at a national level, working to
•HRES 524 EH 
understand DBA’s link to more prevalent disorders fac-
ing Americans; 
(4) commends the Daniella Maria Arturi Founda-
tion 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 success-
ful multidisciplinary approach between clinical and sci-
entific DBA efforts with the goal of shortening the life 
cycle of success realized between the laboratory and ap-
plied patient care; and 
(5) encourages research efforts to further under-
stand ribosomal protein deficiencies in rare inherited dis-
eases and to advance the treatment options available to 
those with DBA. 
Attest: 
Clerk.