- About this Module
- Overview of Thalassemia
- History of Medicine
- Eponyms
- Evolutionary Considerations
- Definitions
- Classification
- Pathophysiology
- Clinical Presentation
- Complications
- Diagnosis
- Treatment
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- Beyond the Guidelines
- References

Long-term efficacy and safety of luspatercept for the treatment of anaemia
Lancet Haematol. 2025 Mar;12(3):e180-e189

A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
N Engl J Med. 2020 Mar 26;382(13):1219-1230

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
N Engl J Med. 2024;390:1663-76

Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassemia
Lancet Haematol. 2022 Oct;9(10):e733-e744

Mitapivat in adults with non-transfusion-dependent thalassemia
Lancet. 2025 Jul 5;406(10498):33-42

Betibeglogene autotemcel gene therapy in patients with transfusion-dependent β-thalassemia
Lancet. 2024;404:2175–86

Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia
N Engl J Med. 2022 Feb 3;386(5):415-27

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
N Engl J Med. 2018 Apr 19;378(16):1479