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Atovaquone is active against AML by upregulating the integrated stress pathway and suppressing oxidative phosphorylation.
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
Paroxysmal cold hemoglobinuria successfully treated with complement inhibition.
Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia.
Related and unrelated donor transplantation for ß-thalassemia major: results of an international survey.
Daratumumab in life-threatening autoimmune hemolytic anemia following hematopoietic stem cell transplantation.
Application of a radiosensitivity flow assay in a patient with DNA ligase 4 deficiency.
Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine.
The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia.
Neurologic event-free survival demonstrates a benefit for SCID patients diagnosed by newborn screening.