Exploring equitable treatment options for pediatric patients with sickle cell disease

Patients with sickle cell disease (SCD) often face a reduced quality of life and a lower life expectancy. Allotransplantation, the first treatment for SCD with curative potential, comes with risks, including transplant-related mortality. Gene therapy, once approved for SCD, could also offer a lifelong cure without the risk associated with allotransplantation.