特罗氟尼定治疗雷特综合征：经验教训。
Trofinetide treatment for Rett syndrome: Lessons to learn.
机构信息
Department 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.
出版信息
Med. 2024 Oct 11;5(10):1194-1196. doi: 10.1016/j.medj.2024.07.028.
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. While further research is needed to fully resolve the condition, insights from trofinetide trials can inform strategies for future treatments and trials.
美国食品和药物管理局（FDA）批准曲非尼治疗雷特综合征，这是第一种改善雷特综合征症状的药物，标志着具有广泛意义的重大里程碑，这对各种疾病都有影响。LILAC 试验证明了曲非尼的长期安全性和有效性。虽然还需要进一步的研究来全面解决这种情况，但曲非尼试验的结果可以为未来的治疗和试验策略提供信息。
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