FDA approves gene therapy for sickle cell disease in children as young as 2 years old
The FDA has approved a gene therapy for sickle cell disease in children as young as 2 years old, expanding access previously limited to those 12 and older. The treatment involves gene-editing a patient's stem cells to produce fetal hemoglobin, offering a potential cure without requiring a donor match.
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- Virginia budget expands Medicaid access to sickle cell gene therapy