[Comment] Mitapivat for transfusion-dependent α-thalassaemia and β-thalassaemia

Thalassaemias are the most common monogenic inherited diseases and are characterised by reduced production of globin chains of haemoglobin. The two most common forms are α-thalassaemia and β-thalassaemia, which affect the production of α-globin and β-globin genes, respectively.1,2 Individuals with severe forms of thalassaemia receive lifelong chronic red blood cell transfusions, resulting in iron overload that leads to severe complications such as endocrinopathy, cardiomyopathy, diabetes, auditory abnormalities, osteoporosis, and growth failure, which can substantially affect their quality of life.