期刊论文Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia复制

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H Ping, R Ding, C Huang, Y Peng…
Zhonghua yi xue yi …, 2026
pubmed.ncbi.nlm.nih.gov
Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures, vosoritide has demonstrated favorable efficacy and a reassuring safety profile. Nevertheless, existing international ACH guidelines largely emphasize complication management and symptomatic care, and there is no unified …