Casgevy has a clear benefit in general health, physical, emotional, social and functional well-being. It potentially provides a one-time functional cure.
The imperative now is development of effective and safe therapies that are readily accessible to the many patients who could benefit.
Benefits from the therapy, called Casgevy, can last for at least five years in people with sickle-cell disease and β-thalassaemia.
Both β-thalassaemia and sickle-cell disease are caused by mutations in genes that code for haemoglobin, which can be compensated by a fetal form.
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