References

Thalassaemia

10.1038/s41572-026-00722-w · 2026 · External reference

The alpha thalassaemias

2009 · External reference

Alpha-thalassemia: a practical overview

10.1016/j.blre.2023.101165 · 2024 · External reference

Pretransfusion hemoglobin level and mortality in adults with transfusion-dependent beta-thalassemia

10.1182/blood.2023022460 · 2024 · External reference

A phase 2 study of luspatercept in adults and adolescents with α-thalassemia: findings from the dose-confirmation cohort in adolescents

2025 · External reference

Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial

10.1016/s0140-6736(25)00635-x · 2025 · External reference

ENERGIZE-T: a global, phase 3, double-blind, randomized, placebo-controlled study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia

10.1182/blood-2024-200867 · 2024 · External reference

Etavopivat improves hemoglobin levels in adults and adolescents with non-transfusion-dependent alpha- or beta-thalassaemia: results from the open-label phase 2 GLADIOLUS study

2026 · External reference

First-in-human study of autologous HBA2-edited CD34+ hematopoietic stem and progenitor cells in alpha-thalassemia with Constant Spring mutation

10.1182/blood-2024-206807 · 2024 · External reference

In utero hematopoietic cell transplantation in fetuses with alpha thalassemia major: a phase 1 clinical trial

10.1182/bloodadvances.2026019655 · 2026 · External reference