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Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2–4 years with Rett syndrome

4 Mar 2025

Abstract excerpt

BACKGROUND: Trofinetide is the first available treatment for Rett syndrome (RTT) and is approved in the United States in adults and pediatric patients aged ≥2 years. The DAFFODIL study was conducted in girls aged 2-4 years with RTT to examine the safety, tolerability, and efficacy of trofinetide and to validate that the recommended dosage, according to body weight, achieved target exposure. METHODS: DAFFODIL was...

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PMID: 40043705
DOI: 10.1016/j.medj.2025.100608

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