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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Authors

Rayan Bou-Fakhredin - Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy

Irene Motta - Department of Internal Medicine, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy

Maria Domenica Cappellini - Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy; Department of Internal Medicine, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy

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