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Blood|September 2, 2009
Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injuryThomas V Adamkiewicz, Miguel R Abboud, Carole Paley, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 30, 2021
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathiesLaura Breda, Valentina Ghiaccio, Naoto Tanaka, 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.
The New England Journal of Medicine|December 10, 2021
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-ThalassemiaFranco Locatelli, Alexis A Thompson, Janet L Kwiatkowski, et al.
Blood|January 12, 2026
Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemiaJanet L Kwiatkowski, Alexis A Thompson, Jennifer Schneiderman, 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|April 24, 2024
Exagamglogene Autotemcel for Transfusion-Dependent β-ThalassemiaFranco Locatelli, Peter Lang, Donna Wall, et al.
American Journal of Hematology|February 12, 2020
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiencyPaola Bianchi, Elisa Fermo, Kimberly Lezon-Geyda, 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.
Haematologica|July 21, 2018
The phenotypic spectrum of germline YARS2 variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2Lisa G Riley, Matthew M Heeney, Joëlle Rudinger-Thirion, et al.
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