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The Journal of Allergy and Clinical Immunology|December 1, 2022
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 DefinitionsChristopher C Dvorak, Elie Haddad, Jennifer Heimall, et al.
The New England Journal of Medicine|June 15, 2022
Sequential Stem Cell-Kidney Transplantation in Schimke Immuno-osseous DysplasiaAlice Bertaina, Paul C Grimm, Kenneth Weinberg, et al.
Blood Advances|August 27, 2025
Improvements in Health-Related Quality of Life in Patients with Transfusion-Dependent β-Thalassemia After Exagamglogene AutotemcelJosu de la Fuente, Haydar Frangoul, Peter Lang, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|January 7, 2014
Avascular necrosis of bone after allogeneic hematopoietic cell transplantation in children and adolescentsXiaxin Li, Ruta Brazauskas, Zhiwei Wang, et al.
American Journal of Hematology|June 8, 2026
Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-ThalassemiaSujit Sheth, Selim Corbacioglu, Josu de la Fuente, et al.
The New England Journal of Medicine|October 5, 2017
Hematopoietic Stem-Cell Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, Christine Duncan, Patricia L Musolino, et al.
The New England Journal of Medicine|April 24, 2024
Exagamglogene Autotemcel for Transfusion-Dependent β-ThalassemiaFranco Locatelli, Peter Lang, Donna Wall, et al.
The New England Journal of Medicine|April 25, 2024
Exagamglogene Autotemcel for Severe Sickle Cell DiseaseHaydar Frangoul, Franco Locatelli, Akshay Sharma, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|June 28, 2015
Second Allogeneic Hematopoietic Cell Transplantation for Patients with Fanconi Anemia and Bone Marrow FailureMouhab Ayas, Mary Eapen, Jennifer Le-Rademacher, et al.
The Lancet. Haematology|February 8, 2024
Diagnosis and management of pyruvate kinase deficiency: international expert guidelinesHanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, et al.
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