Trofinetide treatment for Rett syndrome: Lessons to learn
1Department of Psychiatry, Trinity College Dublin, School of Medicine, Trinity Translational Medicine Institute, Trinity Center for Health Sciences, St James Hospital, D08W9RT Dublin, Ireland; Trinity College Institute of Neuroscience, Trinity College Dublin, Lloyd Building, Dublin 2, Ireland; FutureNeuro, The SFI Research Centre for Chronic and Rare Neurological Diseases, Dublin, Ireland.
None:
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.
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