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A calibrated measuring device (eg, oral syringe, oral dosing cup) should be obtained from the pharmacy to measure and deliver the prescribed dose accurately. 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). The dosage is determined by patient weight and can be administered orally or via gastrostomy tube.

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