[To learn English]
The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals' gene therapy in children as young as two with inherited blood disorders, including sickle cell disease, the first such treatment cleared for this age group.
Casgevy, a one-time treatment made from a patient's own blood stem cells, was previously approved for patients aged 12 and older with sickle cell disease or transfusion-dependent beta thalassemia.
美国食品药品监督管理局批准扩大Vertex制药基因疗法在低至两岁儿童中的使用，这些儿童患有遗传性血液疾病，包括镰状细胞病，这是首个获批用于该年龄段的此类治疗。
Casgevy是一种利用患者自身血液干细胞制成的一次性疗法，此前已被批准用于12岁及以上患有镰状细胞病或输血依赖型β地中海贫血的患者。
LearnEnglish Leave a remark