首个雷特综合征治疗药物trofinetide
Trofinetide;the first drug for the treatment of Rett syndrome
陈静 1罗颖 1陈寅波 1刘相奎1
作者信息
- 1. 中国医药工业研究总院有限公司,上海 201203
- 折叠
摘要
trofinetide是胰岛素样生长因子1(insulin like growth factor-1,IGF-1)的氨基末端三肽甘氨酸-脯氨酸-谷氨酸的合成类似物,于2023年3月10日被美国FDA批准上市,用于治疗2岁及以上成人和儿童患者的Rett综合征.trofinetide是第一种被批准用于治疗Rett综合征的药物,本文对其作用机制、药效学、药动学、临床评价、安全性进行综述.
Abstract
Trofinetide is a synthetic analog of glycine-proline-glutamate which is the N-terminal tripeptide derivative of insulin like growth factor-1(IGF-1).It was approved in march 2023 in the USA for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older.Trofinetide is the first drug approved for the treatment of Rett syndrome,and this article reviews the mechanism of action,pharmacodynamics,pharmacokinetics,clinical evaluation,and safety of trofinetide.
关键词
trofinetide/Rett综合征/胰岛素样生长因子1Key words
trofinetide/Rett syndrome/insulin-like growth factor 1引用本文复制引用
出版年
2024
NETL
NSTL
万方数据