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

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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

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