Sickle cell disease is the most common severe monogenic disorder globally, affecting approximately 300,000 newborns annually, predominantly in sub-Saharan Africa. Hydroxyurea, transfusion therapy, and stem cell transplantation have been established for decades; gene therapy and gene editing approaches have now achieved regulatory approval, offering the prospect of functional cure. Voxelotor and crizanlizumab have expanded the pharmacological toolkit for reducing vaso-occlusive crises.
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