ArticleJournal of the Endocrine Society2026
Effect of vosoritide on spine morphology in children with achondroplasia: 1-year results from a randomized phase 2 study.
Article in Journal of the Endocrine Society, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03583697 (A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months), which is not on this map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months
Who cites it
1 citing paper in PubMed.
- Navepegritide in children with achondroplasia.Translational pediatrics · 2026Article
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Authors and funding
8 authors.
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No grant is acknowledged in the PubMed record.
Abstract
Achondroplasia is a skeletal dysplasia condition caused by reduced endochondral ossification resulting in disproportionate short stature and skeletal deformities, including thoracolumbar kyphosis (TLK) and spinal stenosis. Vosoritide, the first and only approved targeted therapy for achondroplasia, increases bone growth, but its impact on spinal morphology has not been assessed. The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years. Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment. Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model. Measurable improvements in IPD and spinal canal width across L1 to L5 were observed with vosoritide compared with placebo after 1 year. L4 was the most impacted by vosoritide for IPD (LSM difference [95% CI], 0.509 [-0.034 to 1.052] mm,
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