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The dosage is determined by patient weight and can be administered orally or via gastrostomy tube. The most common adverse reactions reported 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 most common adverse reactions reported 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). Rett syndrome in adult and pediatric patients 2 years of age and older.

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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. Having a therapy that has been shown to address multiple symptoms of Rett syndrome provides a promising treatment option that may lead to meaningful impact for patients and their families.

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