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Updated: Oct 15, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Advancing the care of β-thalassaemia patients with novel therapies
Rayan Bou-Fakhredin1, Irene Motta2, Maria Domenica Cappellini1,2
1Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy.
Abstract:
The β-thalassaemias are a group of inherited disorders of haemoglobin synthesis characterised by chronic anaemia of varying severity. Currently available conventional therapies in thalassaemia have many challenges and limitations. A better understanding of the pathology of β-thalassaemia has led to the development of new treatment options, most of which are currently in clinical trials. These could have the potential of reducing red blood cell transfusion burden, raising haemoglobin levels, and improving patients' overall quality of life. In this review, we will provide an overview of the novel therapeutic approaches that are currently under development to advance the care of β-thalassaemia patients.
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