Journal article
International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia
R Savarirayan, J Hoover-Fong, K Ozono, P Backeljauw, V Cormier-Daire, K DeAndrade, P Ireland, M Irving, J Llerena Junior, M Maghnie, M Menzel, N Merchant, K Mohnike, SN Iruretagoyena, K Okada, SO Fredwall
Nature Reviews Endocrinology | Published : 2025
Abstract
Achondroplasia is the most common genetic form of short-limbed skeletal dysplasia (dwarfism). Clinical manifestations and complications can affect individuals across the lifespan, including the need for adaptations for activities of daily living, which can affect quality of life. Current international guidelines focus on symptomatic management, with little discussion regarding potential medication, as therapeutic options were limited at the time of their publication. Vosoritide is the first pharmacological, precision treatment for achondroplasia; it was approved for use in 2021, creating a need for vosoritide treatment guidelines to support clinicians. An international collaborative of leadi..
View full abstractGrants
Funding Acknowledgements
BioMarin, the manufacturer of vosoritide, provided financial support for this project. Logistical support for the consensus building meeting, the Delphi panel and subsequent preparation of the manuscript (that is, compilation of authors' edits, writing style advice and formatting to journal specifications) were provided by R. Kershaw and G. Miles of Prime Access (a division of Prime, Knutsford, UK), who obtained funding from BioMarin. The lead author (R.S.) confirms that neither BioMarin nor Prime Access had any influence or input with regard to the deliberations and final content of this Consensus Statement. BioMarin was not involved in the selection of the guideline development group, defining the guideline scope, the voting process, analysis of the results or preparation of the submitted manuscript. No payments or honoraria were made to the authors with respect to preparation of this manuscript. All participants received honoraria from BioMarin for participation in the consensus building meeting and Delphi panel. BioMarin covered travel and accommodation for participation in the consensus building meeting.