Health: Hope for the Youngest: FDA Approves First Gene Therapy for Children with Sickle Cell Disease

In a moment of genuine good news, the FDA has approved Casgevy — the first gene therapy for children as young as two years old living with sickle cell disease. The treatment uses a child's own stem cells, edited to correct the underlying genetic cause of the disease, delivered as a one-time intravenous infusion. In clinical trials involving children aged 5 to 11, eight of nine patients who could be evaluated achieved 12 full months without needing a blood transfusion. For families living with...