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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. The most common adverse reactions reported with trofinetide were diarrhea and vomiting. The dosage is determined by patient weight and can be administered orally or via gastrostomy tube. 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).

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