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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 product, which received FDA approval in March 2023, was approved based on data 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. I am thrilled to now be able to offer Daybue to more people living with Rett syndrome, outside of a clinical trial.

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