FDA drug approval affects health care around the world, but political shortcuts could hurt
At the beginning of July 2026, the U.S. Food and Drug Administration approved the first gene therapy for young children with sickle cell disease.
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FDA expands use of first sickle cell disease gene therapy for children as young as 2 years
The U.S. Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients ages 2 and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT). Supplement