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Exagamglogene autotemcel (Casgevy°) in beta-thalassaemia or sickle-cell disease

 Marketing Authorisations  Exagamglogene autotemcel is the first gene therapy product using the CRISPR-Cas9 system to have been granted marketing authorisation in the European Union. It is indicated in certain patients with severe forms of beta-thalassaemia or sickle-cell disease. Is it curative in these patients? Does it eliminate the need for blood transfusions and iron chelators in severe beta-thalassaemia, does it eliminate vaso-occlusive crises and their complications in sickle-cell disease, and what are its adverse effects?
Full article (4 pages) available for download by subscribers

 ©Prescrire 1 March 2025

Source: "Exagamglogene autotemcel (Casgevy°) in certain patients with severe beta-thalassaemia or severe sickle-cell disease" Prescrire Int 2025; 34 (268): 61-65. Subscribers only.

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See also:

"Betibeglogene
autotemcel - Zynteglo°.
In certain forms of severe
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"Luspatercept (Reblozyl°)
in anaemia associated
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32 (245): 44.
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