周六报道，Vertex宣布对5-11岁儿童进行的CASGEVY研究显示，与12岁及以上儿童相比，具有变革潜力、一致的有效性和安全性

2025-12-08 17:35

- Data from pivotal studies of CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrates the transformative potential of the therapy in younger patients -
- Efficacy and safety data in children 5-11 years are consistent with the durable and positive benefit/risk profile established from clinical studies in patients 12 years of age and older -
- Vertex expects to initiate global regulatory submissions for CASGEVY in children 5-11 years in 1H 2026 -