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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. Findings showed that treatment with trofinetide were diarrhea and vomiting.

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