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Article

Current and emerging therapies for Achondroplasia: The dawn of precision medicine.

Bioorganic & medicinal chemistry - 3 May 2023

Dardenne Etienne, Ishiyama Noboru, Lin Tai-An, Lucas Matthew C

Abstract excerpt

Achondroplasia is a rare disease affecting bone growth and is caused by a missense mutation in the fibroblast growth factor receptor 3 (FGFR3) gene. In the past few years, there were multiple experimental drugs entering into clinical trials for treating achondroplasia including vosoritide, the first precision medicine approved for this indication. This perspective presents the mechanism of action, benefit, and...

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PMID: 37156065
DOI: 10.1016/j.bmc.2023.117275

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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