Main
seizures, hand stereotypies, loss of verbal communication, neurodevelopmental disorder, Rett syndrome, RTT
RETT SYNDROME
Rett syndrome (RTT) is a rare, genetic neurodevelopmental disorder characterized by loss of verbal communication with limited nonverbal skills, loss of fine and gross motor function (including purposeful hand use), behavioral issues, seizures, hand stereotypies and gastrointestinal problemsRTT primarily affects females (1 in 10,000–15,000 live female births)Trofinetide ((2The main objective of this phase 3 study was to investigate the efficacy, safety and tolerability of trofinetide in a larger, randomized, double-blind, placebo-controlled study in RTT.