美国食品药品管理局批准了第一种基因疗法, 用于治疗儿童严重免疫缺陷. Rocket Pharma gets FDA approval for first gene therapy treating severe immune deficiency in children.

罗克特制药公司已获得FDA的加速批准,该疗法是针对重型白细胞粘附缺陷I类第一个基因治疗. Rocket Pharmaceuticals has received FDA accelerated approval for KRESLADI, the first gene therapy for severe leukocyte adhesion deficiency type I. 这种一次性治疗使用患者自己的干细胞恢复免疫功能, 提供了对缺乏匹配捐赠者的儿童潜在的治愈. This one-time treatment uses a patient's own stem cells to restore immune function, offering a potential cure for children lacking matched donors. 虽然长期益处必须得到确认,但早期数据显示感染和住院减少. While long-term benefits must be confirmed, early data shows reduced infections and hospitalizations.