Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.
Daniel G GlazeJeffrey L NeulWalter E KaufmannElizabeth Berry-KravisSean CondonGeorge StomsSean OosterholtOscar Della PasquaLarry GlassNancy E JonesAlan K Percynull nullPublished in: Neurology (2019)
This study provides Class I evidence that for children/adolescents with RTT, trofinetide was safe, well-tolerated, and demonstrated improvement over placebo at 200 mg/kg bid in functionally important dimensions of RTT.