Key clinical point: Trofinetide provides clinically meaningful improvements in core symptoms of Rett syndrome.
Major finding: The 200 mg/kg b.i.d. dose of trofinetide improved outcomes on the Rett Syndrome Behavior Questionnaire by 16%.
Study details: A phase 2, double-blind, placebo-controlled study of 82 children and adolescents with Rett syndrome.
Disclosures: Neuren Pharmaceuticals and Rettsyndrome.org funded the study.
Glaze DG et al. Neurology. 2019 Mar 27. doi: 10.1212/WNL.0000000000007316.