Inherited haemoglobin disorders, including thalassaemia and sickle-cell disease, are the
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …

Abstract β-Thalassemia is caused by reduced (β+) or absent (β 0) synthesis of the β-globin
chains of hemoglobin. Three clinical and hematological conditions of increasing severity are …

Non-transfusion-dependent thalassemias include a variety of phenotypes that, unlike
patients with beta (β)-thalassemia major, do not require regular transfusion therapy for …

Background In patients with non-transfusion-dependent β-thalassaemia, haemoglobin
concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality …

The complex pathophysiology in β-thalassemia can translate to multiple morbidities that
affect every organ system. Improved survival due to advances in management means that …

Recent molecular studies of fetal hemoglobin (HbF) regulation have reinvigorated the field
and shown promise for the development of clinical HbF inducers to be used in patients with …

Abstract β-Thalassemia is one of the most common monogenetic diseases worldwide, with a
particularly high prevalence in the Middle East region. As such, we have developed long …

Our understanding of the molecular and pathophysiological mechanisms underlying the
disease process in patients with β thalassaemia intermedia (TI) has substantially increased …

Background Patients with β thalassemia intermedia can have substantial iron overload,
irrespectively of their transfusion status, secondary to increased intestinal iron absorption …

In β-thalassemia, unequal production of α-and β-globin chains in erythroid precursors
causes apoptosis and inhibition of late-stage erythroid differentiation, leading to anemia …