中国临床药理学杂志2024，Vol.40Issue(18)：2743-2746,4.DOI:10.13699/j.cnki.1001-6821.2024.18.028
治疗Rett综合征的首个新药——Daybue
Daybue,the first new drug for the treatment of Rett syndrome
李妍 1郝晔2
作者信息
- 1. 山西省儿童医院、山西省妇幼保健院药剂科,山西太原 030013
- 2. 南京医科大学附属儿童医院药学部,江苏南京 210007
- 折叠
摘要
Abstract
Daybue oral solution has been approved by the U.S.Food and Drug Administration(FDA)in March 2023 for the treatment of Rett syndrome in adults and children aged two years and above,becoming the first and only drug approved for the treatment of Rett syndrome.The main active component of Daybue is trofinetide,a novel synthetic analogue of the amino terminal tripeptide of insulin-like growth factor Ⅰ(IGF-1).It can stimulate synaptic maturation,overcome synaptic and neuronal immaturity,and treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.This paper introduces it's mechanism of action,pharmacodynamics,pharmacokinetics,clinical research and safety.关键词
Daybue/曲非奈肱/Rett综合征Key words
Daybue/trofinetide/Rett syndrome分类
医药卫生引用本文复制引用
李妍,郝晔..治疗Rett综合征的首个新药——Daybue[J].中国临床药理学杂志,2024,40(18):2743-2746,4.
