CECMED

The U.S. Food and Drug Administration (FDA) has expanded the approved use of gene therapies for sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT), marking a significant shift in treatment options for pediatric patients. These therapies, which utilize a patient’s own modified hematopoietic stem cells, are now accessible to younger populations previously limited by age-based … Read more