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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. Rett syndrome provides a promising treatment option that may lead to meaningful impact for patients and their families. The dosage is determined by patient weight and can be administered orally or via gastrostomy tube.

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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 dosage is determined by patient weight and can be administered orally or via gastrostomy tube. Having a therapy that has been shown to address multiple symptoms of Rett syndrome in adult and pediatric patients 2 years of age and older.

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