thalassemia major
- n a fatal form of homozygous thalassemia (inherited from both parents) in which there is no hemoglobin; skeletal deformations; heart and spleen and liver enlarged
thalassemia major :
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- bluebird bio Announces First Patient Transplanted in Phase 1/2 Northstar Study for the Treatment of Beta-Thalassemia
CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq:BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that the first subject with beta-thalassemia major has been enrolled in its phase 1/2 Northstar Study (HGB-204) in the United States and has undergone infusion with bluebird bio’s ...
March 27, 2014 - Business Wire