Published in Blood Weekly, October 23rd, 1995
(beta)0-thalassemia is a disease characterized by severe anemia that begins during the first month of life. As the level of fetal hemoglobin declines, the affected child is unable to replace it with the major adult hemoglobin.
Individuals become transfusion-dependent, and while transfusion medicine has vastly improved survival over the past few years, significant complications from transfusion associated hepatitis and iron overload remain problematic.
Bone marrow transplantation...
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