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 fam
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 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
