IV 
110TH CONGRESS 
1ST SESSION 
H. RES. 524 
Expressing the sense of the House of Representatives with respect to 
Diamond-Blackfan Anemia. 
IN THE HOUSE OF REPRESENTATIVES 
JUNE 27, 2007 
Mrs. MCCARTHY of New York submitted the following resolution; which was 
referred to the Committee on Energy and Commerce 
RESOLUTION 
Expressing the sense of the House of Representatives with 
respect to Diamond-Blackfan Anemia. 
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 unfair consequences of their rare disease status, 
which often means limited research is being done and 
treatment options may not be optimal; 
Whereas individuals suffering from rare diseases deserve ac-
cess to improved treatment options and the potential for 
VerDate Aug 31 2005 
06:13 Jun 28, 2007
Jkt 059200
PO 00000
Frm 00001
Fmt 6652
Sfmt 6300
E:\BILLS\HR524.IH
HR524
bajohnson on PROD1PC77 with BILLS
•HRES 524 IH 
a cure through the advancement of specialized research 
initiatives despite their rare disease status; 
Whereas it is recognized that although DBA is rare, the im-
portance of the disease lies only in part in the dev-
astating clinical consequences it may have on those af-
fected, as research is proving that study of complex, rare 
diseases such as DBA are yielding tremendous advance-
ments 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 de-
fect; 
Whereas researchers believe that as a genetic disorder of red 
cell production and as a cancer predisposition syndrome 
with a high rate of congenital anomalies, the study of 
DBA will yield clues to several other widespread diseases 
providing valuable insights into the biology of blood dis-
orders, blood cell formation (recovery from cancer chemo-
therapy), 
the 
true 
incidence 
of 
aplastic 
anemia, 
myelodysplastic syndrome, leukemia, and the predisposi-
tion to cancer in DBA, and 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; 
VerDate Aug 31 2005 
06:13 Jun 28, 2007
Jkt 059200
PO 00000
Frm 00002
Fmt 6652
Sfmt 6300
E:\BILLS\HR524.IH
HR524
bajohnson on PROD1PC77 with BILLS
•HRES 524 IH 
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, where there are no re-
gional or ethnic biases and a small number of patients, 
making progress in treatment protocols and research dif-
ficult, 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, which has become the multi-
dimensional hub for the care and treatment of DBA pa-
tients across the country, as well as the home of the 
DBA Patient Registry which has become a valuable na-
tional 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 DBA Com-
prehensive Clinical Care Center at Schneider Children’s 
Hospital 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 DBA Comprehensive Clin-
ical Care Center prompted the Centers for Disease Con-
trol and Prevention’s DBA Public Health Outreach and 
Surveillance Program to establish 3 additional DBA Cen-
ters 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 es-
tablishment; 
VerDate Aug 31 2005 
06:13 Jun 28, 2007
Jkt 059200
PO 00000
Frm 00003
Fmt 6652
Sfmt 6300
E:\BILLS\HR524.IH
HR524
bajohnson on PROD1PC77 with BILLS
•HRES 524 IH 
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 achieved through the federally sup-
ported DBA initiatives within the National Institutes of 
Health and 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 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 
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 
VerDate Aug 31 2005 
06:13 Jun 28, 2007
Jkt 059200
PO 00000
Frm 00004
Fmt 6652
Sfmt 6300
E:\BILLS\HR524.IH
HR524
bajohnson on PROD1PC77 with BILLS
•HRES 524 IH 
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— 
(1) the House of Representatives— 
(A) recognizes that the identification of Di-
amond-Blackfan Anemia (in this resolution re-
ferred to as ‘‘DBA’’) as the first human dis-
order with a ribosomal deficiency is a primary 
example of the importance of the Federal Gov-
ernment’s continued support of DBA research, 
which may advance the understanding of the 
basic mechanisms that affect red cell produc-
tion, identify connections of ribosomal function 
and cell cycle production, identify implications 
of cancer predisposition, and serve as an impor-
tant model for understanding human develop-
ment and the molecular basis for certain birth 
defects; 
(B) recognizes that Federal support of 
comprehensive centers for rare disease patients 
enhances the ability for experienced doctors to 
provide the most complete care for each patient, 
leading to an increase in correct and early diag-
VerDate Aug 31 2005 
06:13 Jun 28, 2007
Jkt 059200
PO 00000
Frm 00005
Fmt 6652
Sfmt 6201
E:\BILLS\HR524.IH
HR524
bajohnson on PROD1PC77 with BILLS