Atlas One · Host Biology · Scale 07 — System

Thalassemia

Thalassaemias are haemoglobinopathies from α- or β-globin chain imbalance; β-thalassaemia major requires lifelong transfusion; ineffective erythropoiesis → iron overload despite anaemia; betibeglogene autotemcel (Zynteglo) and CRISPR-based Casgevy are approved gene therapies.

Also known as: thalassaemia, thalassemia, beta-thalassemia, alpha-thalassemia, β-thalassaemia major, Cooley's anaemia, thal major, HbH disease, hydrops fetalis, thal trait

Scale 07 — SystemScale
draftStatus
2026-06-08Last Reviewed
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thalassemia

Thalassemia

Thalassaemias are haemoglobinopathies from α- or β-globin chain imbalance; β-thalassaemia major requires lifelong transfusion; ineffective erythropoiesis → iron overload despite anaemia; betibeglogene autotemcel (Zynteglo) and CRISPR-based Casgevy are approved gene therapies.