The FDA approved a one-time gene therapy for severe sickle cell disease in patients 12 and older, though cost and access remain major concerns.
The US Food and Drug Administration has approved a one-time gene therapy for sickle cell disease in patients aged 12 and older with recurrent vaso-occlusive crises.
In pivotal trials, the majority of treated patients were free of severe pain crises through the follow-up period. The therapy is administered after a course of conditioning chemotherapy.
Access and cost remain significant concerns, with specialists warning that the number of centres equipped to deliver the treatment is currently limited.
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