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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 product, which received FDA approval in March 2023, was approved based on data from the randomized, double-blind, placebo-controlled phase 3 LAVENDER study (ClinicalTrials. Daybue is supplied as a strawberry-flavored oral solution containing 200 mg of trofinetide per mL. 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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