"Both sickle cell disease and beta-thalassemia are painful, lifelong conditions that in some cases can be fatal," said Julian Beach, interim executive director of healthcare quality and access at the UK's MHRA
"In a trial run by Vertex and Crispr Therapeutics, 45 patients have been treated with Casgevy but only 29 have been followed for at least 18 months. Of those, 28 were free of severe pain crises for at least a year after treatment."
"In a study of beta thalassemia patients, 54 patients have so far received Casgevy. Of 42 that have been followed long enough, 39 did not need a blood transfusion for at least a year after treatment."
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