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Advances in the management of achondroplasia

Nature Reviews Endocrinology (2024)Cite this article

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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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Authors and Affiliations

  1. Murdoch Children’s Research Institute and University of Melbourne, Parkville, Victoria, Australia

    Ravi Savarirayan

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  1. Ravi Savarirayan
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Correspondence to Ravi Savarirayan.

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Competing interests

R.S. has received honoraria from BioMarin, QED pharma, Ascendis and Sanofi.

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Savarirayan, R. Advances in the management of achondroplasia. Nat Rev Endocrinol (2024). https://doi.org/10.1038/s41574-024-00994-w

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