曲非奈德治疗雷特综合征：经验教训。

IF 12.8 Q1 MEDICINE, RESEARCH & EXPERIMENTAL

Med Pub Date : 2024-10-11 DOI:10.1016/j.medj.2024.07.028

Daniela Tropea

引用次数: 0

摘要

美国食品和药物管理局（FDA）批准特罗非肽作为首个改善雷特综合征症状的药物疗法，这是一个重要的里程碑，对各种疾病具有广泛的影响。LILAC试验证明了特罗芬肽的长期安全性和有效性1,2。虽然要彻底解决这一病症还需要进一步的研究，但特罗芬肽试验的启示可以为未来的治疗和试验提供参考。

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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.

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来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

期刊介绍： Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically. Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.