软骨发育不全的治疗进展

IF 3.7 3区医学 Q2 CHEMISTRY, MEDICINAL

Chemical Research in Toxicology Pub Date : 2024-05-13 DOI:10.1038/s41574-024-00994-w

Ravi Savarirayan

引用次数: 0

摘要

首个针对软骨发育不全儿童的精准疗法--沃索利肽现已在澳大利亚、美国、欧盟和日本等多个地区获得批准。本文将讨论该疗法在生长发育和软骨发育不全相关并发症方面的最新试验结果，并探讨其临床应用的合理性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Advances in the management of achondroplasia

The first precision therapy for children with achondroplasia, vosoritide, is now approved in many regions, including Australia, the USA, the European Union and Japan. This article discusses the recent trial results for this therapy regarding growth and the co-morbidities associated with achondroplasia and considers the rationale for its clinical use.

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来源期刊

Chemical Research in Toxicology 医学-毒理学

CiteScore

7.90

自引率

7.30%

发文量

215

审稿时长

3.5 months

期刊介绍： Chemical Research in Toxicology publishes Articles, Rapid Reports, Chemical Profiles, Reviews, Perspectives, Letters to the Editor, and ToxWatch on a wide range of topics in Toxicology that inform a chemical and molecular understanding and capacity to predict biological outcomes on the basis of structures and processes. The overarching goal of activities reported in the Journal are to provide knowledge and innovative approaches needed to promote intelligent solutions for human safety and ecosystem preservation. The journal emphasizes insight concerning mechanisms of toxicity over phenomenological observations. It upholds rigorous chemical, physical and mathematical standards for characterization and application of modern techniques.