Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results from the Open-Label LILAC and LILAC-2 Studies

Kurt Huber; Alan K. Percy; Jeffrey L. Neul; Tim A. Benke; Elizabeth Berry‐Kravis; Daniel G. Glaze; Eric D. Marsh; Kathie M. Bishop; James M. Youakim

doi:10.1055/s-0045-1812170

September 26, 2025Neuropediatrics

Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results from the Open-Label LILAC and LILAC-2 Studies

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Key Points

Trofinetide shows long-term safety and efficacy in treating Rett syndrome, suggesting a positive outcome for patients.
In the LILAC studies, trofinetide significantly improved core symptoms of Rett syndrome, which points to its potential as a valuable treatment option.
Open-label extension studies, such as LILAC and LILAC-2, provide real-world evidence of trofinetide's beneficial effects in patients with Rett syndrome.
The safety profile of trofinetide in the LAVENDER study indicates it may be well-tolerated among patients with Rett syndrome, suggesting a supportive treatment choice.

Implication

Open-label studies revealed improved efficacy and safety of trofinetide in Rett syndrome, highlighting its potential benefits.

Abstract

Background/Purpose: Trofinetide significantly improved core symptoms of Rett syndrome (RTT) with an acceptable safety profile in LAVENDER. Here, we report the safety and efficacy results of LILAC and LILAC-2, open-label extension studies of LAVENDER.

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Cite This Study

Huber et al. (2025) studied this question.

synapsesocial.com/papers/68d90a0a41e1c178a14f6608 https://doi.org/10.1055/s-0045-1812170

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Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results from the Open-Label LILAC and LILAC-2 Studies

Key Points

Implication

Abstract

Discussion

Cite This Study

Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results from the Open-Label LILAC and LILAC-2 Studies

Key Points

Implication

Abstract

Discussion

Cite This Study

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