Trofinetide treatment for Rett syndrome: Lessons to learn.

The US FDA approval of trofinetide as the first pharmacological treatment to improve Rett syndrome's symptomatology marks a significant milestone with broad implications for various disorders. The LILAC trials demonstrate long-term safety and efficacy of trofinetide. ^{1 , 2} While further research is needed to fully resolve the condition, insights from trofinetide trials can inform strategies for future treatments and trials.
Competing Interests: Declaration of interests D.T. is a consultant for Acadia through Trinity Consult. Possible earnings from this position is devoted to research. D.T. is one of the authors in the U.S. Patent: WGS Ref. No. M0656.70145US01 – “Treatment of Rett syndrome and other disorders.” However, D.T. has no financial benefits from this patent.
(Copyright © 2024 Elsevier Inc. All rights reserved.)