ArticleJournal of the Endocrine Society2026
Real-world growth outcomes of vosoritide in children with achondroplasia: a single-center cohort 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. Cited by 1 paper.
What it found
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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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Navepegritide in children with achondroplasia.Translational pediatrics · 2026Article
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Context: Vosoritide, a C-type natriuretic peptide analogue, is the first approved therapy targeting the pathophysiology of achondroplasia. While clinical trials demonstrate efficacy, individuals with prior orthopedic surgeries are typically excluded, and real-world data remain limited. Objective: To evaluate real-world growth outcomes of vosoritide therapy in children with achondroplasia, including those with prior surgical interventions. Methods: This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center. Z-scores were calculated using an AI-assisted growth assessment tool previously validated for children with achondroplasia. Δ z-scores for height, sitting height, arm span, and body mass index (BMI) were calculated from baseline to follow-up. Multiple linear regression analyses assessed predictors of response. Results: Mean height z-scores improved from -0.62 ± 1.09 to -0.24 ± 1.20 ( Conclusion: Vosoritide therapy enhanced linear and appendicular growth in a real-world cohort of children with achondroplasia, including post-surgical patients. These findings align with clinical trial data and support the therapy's applicability across diverse clinical profiles.
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