MHRA approves gene-editing treatment for children aged five and over with serious inherited blood disorders

Synopsis

MHRA approves gene-editing treatment for children aged five and over with serious inherited blood disorders Children aged five and over with severe sickle-cell disease and transfusion-dependent β-thalassemia, can now be treated with Casgevy (exagamglogene autotemcel), following an extension of its UK authorisation by the MHRA.

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