FDA approves first gene therapy for children with sickle cell disease

thehill.com

FDA approves Casgevy, the first gene therapy for children aged 2 and older with sickle cell disease. This one-time intravenous infusion uses a patient's own stem cells to treat the debilitating and life-threatening condition. Sickle cell disease affects red blood cells, causing pain and complications. The therapy offers a chance for a healthier future.


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FDA approves first gene therapy for children with sickle cell disease | News Minimalist