1. Implications of Emerging Treatments for Beta-Thalassemia

3. Introduction

4. Thalassemia Continues to Be an Evolving Global Burden due to Migration

5. The Pathophysiology of Disease

6. Hallmark of Disease in Thalassemia

7. The Role of Transfusion and the Associated Challenges

8. Evidence for Iron Chelation Therapy in TDT Is Established

9. Treatment Options for Thalassemia

10. Gene Therapy in Thalassemia: Patients Who Stopped Transfusions

11. Gene Therapy in Thalassemia: Changes in the Number of Transfusions

12. Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia

13. Treatment Options for Thalassemia (cont)

14. Luspatercept

15. BELIEVE Trial: Study Design

16. BELIEVE Trial: Rate of Erythroid Response

17. BELIEVE Trial: Secondary Endpoints

18. BELIEVE Trial: Reduction in Transfusion Burden During Any 12- and 24-Week Interval

19. Potential Impact of the BELIEVE Trial Efficacy Results

20. BELIEVE Trial: Safety

21. Potential Implications of the Safety Findings from the BELIEVE Trial

22. Novel Agents and Their Mechanisms of Action

23. Novel Therapy in Thalassemia

24. Hydroxyurea and Bitopertin

25. Novel Therapy in Thalassemia (cont)

26. Novel Therapy in Thalassemia (cont)

27. Novel Therapies Targeting Iron Dysregulation Are Under Development

28. Considerations for the Future

29. Abbreviations

30. Abbreviations (cont)