A New Hope in the Treatment of Rett Syndrome: Trofinetide

Trofinetide is a medication used to treat Rett Syndrome. It was approved by the United States in March 2023 for the treatment of Rett Syndrome in adults and children ages 2 and older. The drug is an N-terminal tripeptide derivative of insulin-like growth factor-1 (IGF-1) and is an oral, small molecule, synthetic analogue. The FDA\'s approval of trophinetide marks the first authorized treatment for Rett Syndrome.