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Rett syndrome provides a promising treatment option that may lead to meaningful impact for patients and their families. Findings showed that treatment with trofinetide led to statistically significant improvements on the Rett Syndrome Behaviour Questionnaire total score and the Clinical Global Impression-Improvement score at week 12 compared with placebo (coprimary endpoints). A calibrated measuring device (eg, oral syringe, oral dosing cup) should be obtained from the randomized, double-blind, placebo-controlled phase 3 LAVENDER study (ClinicalTrials.

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