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1. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines

3. Lovotibeglogene Autotemcel Gene Therapy for Sickle Cell Disease: 60 Months Follow-up

6. Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial

7. Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

9. Efficacy and Safety in Patients (Pts) with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

10. Transfusion Independence after Exagamglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia

11. Outcomes of Unrelated Partial T Cell Depleted Stem Cell Transplantation for Patients with Hemoglobinopathies

12. 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)

14. List of contributors

15. Transcranial doppler re‐screening of subjects who participated in STOP and STOP II

17. Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent, Severe Genotype β‑Thalassaemia: Results from the HGB-212 (Northstar-3) Study

18. Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

21. Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open‐label FIRST study

23. Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study

24. Zinc supplementation improves bone density in patients with thalassemia: a double-blind, randomized, placebo-controlled trial 1 , 2 , 3

25. Increased leucocyte apoptosis in transfused β‐thalassaemia patients

28. 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

29. Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease (SCD): Updated Group C Clinical Results and Investigations into Two Cases of Anemia from the Phase 1/2 HGB-206 Study

30. Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia—TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial

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

34. Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

35. 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

38. Contributors

39. Increased prevalence of potential right‐to‐left shunting in children with sickle cell anaemia and stroke

42. Sustained Improvements in Patient Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

43. Efficacy and Safety of Betibeglogene Autotemcel (beti-cel) Gene Therapy in 63 Patients with Transfusion-Dependent β-Thalassemia (TDT): 7-Year Post-Infusion Follow-up of Phase 1/2 and Phase 3 Studies

44. Polyclonality Strongly Correlates with Biologic Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of Lentiglobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

45. Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

46. Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-Thalassemia

47. List of Contributors

49. Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report

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