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April 9, 2024Neurology

Trofinetide for the Treatment of Girls Aged Two to Four Years with Rett Syndrome: Final Results from the Open-label DAFFODIL Study (S37.003)

Alan K. Percy Robin Ryther Eric D. Marsh Timothy Feyma David N. Lieberman Jeffrey L. Neul Tim A. Benke Daniel G. Glaze Elizabeth Berry‐Kravis Amitha Ananth Colleen Buhrfiend Di An Mona Darwish Dilesh Doshi Kathie Bishop James M. Youakim

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Abstract

To investigate the safety/tolerability and exploratory efficacy of long-term treatment with trofinetide in girls aged 2–4 years with Rett syndrome (RTT).

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Cite This Study

Percy et al. (2024) studied this question.

synapsesocial.com/papers/68e6fca8b6db643587676698 https://doi.org/10.1212/wnl.0000000000204788

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results from the Open-Label LILAC and LILAC-2 Studies2025
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4Efficacy and Safety of Trofinetide for the Treatment of Rett Syndrome: Results from the Pivotal Phase 3 LAVENDER Study2025
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