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A subsequent double-blind, randomized, placebo-controlled study of trofinetide in pediatric patients with Rett syndrome demonstrated that trofinetide at 200 mg/kg twice a day showed statistically significant and clinically relevant improvements relative to placebo on the Rett Syndrome Behavior Questionnaire, Rett Syndrome-Clinician Domain Specific Concerns-Visual Analog Scale, and Clinical Global Impression Scale-Improvement (96)

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The mitochondrial-enriched fraction was treated with 200 M JC-1 and incubated at 37 C for 30 min

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