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3. Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial

4. Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency

5. Sustained Efficacy and Safety in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

6. Mechanisms of plasma non‐transferrin bound iron generation: insights from comparing transfused diamond blackfan anaemia with sickle cell and thalassaemia patients

10. S273: LONG-TERM ERYTHROID RESPONSE DATA FROM PATIENTS (PTS) WITH NON-TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA (NTDT) RECEIVING LUSPATERCEPT IN THE BEYOND TRIAL

12. P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED

13. SLN124, a GalNAc conjugated 19‐mer siRNA targeting tmprss6, reduces plasma iron and increases hepcidin levels of healthy volunteers

16. Health‐related quality of life in patients with β‐thalassemia: Data from the phase 3 BELIEVE trial of luspatercept

17. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency

18. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency

23. Long-term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed-up to 7 Years after Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy (GT) and Factors Impacting Neutrophil and Platelet Engraftment

24. Early‐onset reduced bone mineral density in patients with pyruvate kinase deficiency

31. Effect of Luspatercept in β-Thalassemia Patients with β0/β0 Genotype: A Subgroup Analysis of the BELIEVE Study

32. Investigations into the Mechanisms and Clinical Implications of Modulation of Hepcidin Levels By Luspatercept in TD MDS and TD b-Thalassemia

33. Effect of Luspatercept on Red Blood Cell (RBC) Transfusion Burden, Iron Chelation Therapy (ICT), and Iron Overload in Adults with Transfusion-Dependent β-Thalassemia (TDT) from the BELIEVE Trial: A Long-Term Analysis

35. A Closer Look at Changes in Hemoglobin Levels in Patients with Non-Transfusion Dependent β-Thalassemia Treated with Luspatercept: Post Hoc Analysis of the Phase 2 BEYOND Trial

36. Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

37. Erythroid Response in Patients with Non-Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Long-Term Data from the BEYOND Trial

38. Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

39. Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

40. Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial

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