Findings support mitapivat as the first oral disease-modifying therapy for adults with transfusion-dependent α-thalassaemia or β-thalassaemia, providing a new treatment option to reduce transfusion burden in this patient population.
M. Cappellini, Sujit Sheth, A. Taher et al.· The Lancet· 3 citations
The emerging role of repurposed low-cost therapies, particularly hydroxyurea and thalidomide, as disease-modifying options in resource-limited settings are discussed and opportunities to democratise access to curative gene therapies through technological developments, decentralised manufacturing, regional collaboration...
Hanny T. Al-Samkari, F. Aboobacker, B. Andemariam et al.· The Lancet Haematology· 2 citations
Non-transfusion-dependent thalassemia (NTDT) describes a group of inherited hemoglobin disorders characterized by hemolytic anemia and ineffective erythropoiesis. Although patients with NTDT do not require lifelong regular transfusions for survival, they are at a substantial risk of complications including iron overloa...
Aaron N. Cheng, Hanny T. Al-Samkari· Therapeutic delivery· 0 citations
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