News: FDA Approves Gene Therapy for Children with Sickle Cell Disease : A Medical Milestone

In a major medical breakthrough, the U.S. Food and Drug Administration has approved Casgevy, the first gene therapy for children as young as two years old living with sickle cell disease. The one-time intravenous infusion uses the patient's own stem cells to address the root cause of the disorder, offering the possibility of long-term relief from a condition that affects millions worldwide. Clinical data from patients aged 5 to 11 showed transfusion independence for 12 months in the majority...