The purpose of this article is to set forth our approach to diagnosing and managing the
thalassemias, including β-thalassemia intermedia and β-thalassemia major. The article …

β-Thalassemia, which is caused by a decrease in the production of β-globin chains, affects
multiple organs and is associated with considerable morbidity and mortality. Lifelong care is …

Thalassemia major and sickle cell disease are the two most widely disseminated hereditary
hemoglobinopathies in the world. The outlook for affected individuals has improved in recent …

Background Failure to generate phagocyte-derived superoxide and related reactive oxygen
intermediates (ROIs) is the major defect in chronic granulomatous disease, causing …

The Sickle Cell Unrelated Donor Transplant Trial (SCURT trial) of the Blood and Marrow
Transplant Clinical Trials Network (BMT CTN) is a phase II study of the toxicity and efficacy …

The thalassemia syndromes are hemoglobin disorders that result from significantly reduced
or absent synthesis of either the α-or β-globin chains. The result is a chronic hemolytic …

Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic
hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa …

Graft failure is a significant complication following allogeneic hematopoietic cell
transplantation (alloHCT). It may result from rejection caused by recipient T cells, natural …

The treatment of the more than 1,800 known monogenic hereditary disorders will depend on
the development of'genetic medicines'—therapies that use the transfer of DNA and/or RNA …

Beta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of little or
no β-globin chains. Novel approaches are being developed to correct the resulting α/β …