P271: Long-term vosoritide improves bone mineral density and bone content in children with achondroplasia: Results from quantitative computed tomography analyses

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Authors: Klaus Engelke, Thomas P. Fuerst, Cheng-Chia Tang, Andrea Löw, Jeanette White
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Abstract

Vosoritide, a C-type natriuretic peptide analogue that stimulates endochondral bone growth, is the only approved precision therapy for children with achondroplasia (ACH). The ongoing CANOPY ACH clinical program has over a decade of longitudinal data demonstrating that vosoritide is well tolerated and causes significant, sustained improvements in annualized growth velocity. The effect of long-term vosoritide use on bone has not been investigated. We used peripheral quantitative computed tomography (QCT) imaging to assess bone growth parameters in children with ACH using vosoritide long-term.

Fields of Study & Subjects

Connective tissue disorders research Growth Hormone and Insulin-like Growth Factors Bone health and osteoporosis research

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103765–103765
Publication date
1 January 2026
Language
EN