Latest / This Week In Medicine
NEJM 9 10 2026 Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease
This study investigates a breakthrough gene-editing treatment known as exa-cel, which modifies a patient’s own blood-building cells to stimulate the production of fetal hemoglobin. By targeting children as young as five who suffer from sickle cell disease or transfusion-dependent β-thalassemia, researchers aimed to eliminate the most debilitating symptoms of these genetic disorders. The clinical results were remarkable, as every evaluable participant achieved complete freedom from blood transfusions or painful vascular blockages for over a year. Despite these clinical triumphs, the process…
The skinny
The skinny isn't ready yet — notes appear once the transcript is processed.