1.
Indian Pediatr
;
2023 Apr; 60(4): 313-316
Article
| IMSEAR
| ID: sea-225410
ABSTRACT
The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with ?-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of ?-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy