Trofinetide;the first drug for the treatment of Rett syndrome
CHEN Jing
LUO Ying
CHEN Yin-bo
LIU Xiang-kui
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.
Keywords：trofinetideRett syndromeinsulin-like growth factor 1
Publication Date：2024-09-15
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：5( 1756-1760 )