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.
Trofinetide;the first drug for the treatment of Rett syndrome
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.
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