ArticleJournal of the Endocrine Society2025
Real-world Outcome of Vosoritide Treatment in Children With Achondroplasia: A 12-month Retrospective Observational Study.
Article in Journal of the Endocrine Society, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
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
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Efficacy and safety of vosoritide in children with achondroplasia: a systematic review and meta-analysis.European journal of pediatrics · 2026Pooled it
- Real-World Outcomes of Vosoritide Treatment in Chinese Children with Achondroplasia: The Retrospective Cohort COREV Study.Advances in therapy · 2026Observational
- Early closure of cranial base synchondroses in infants with Vosoritide-treated achondroplasia.Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology · 2026Article
- Healthcare and Psychosocial Needs in Achondroplasia Across the Lifespan: Developmental Functioning, Multidisciplinary Care, and Family-Centered Outcomes.Healthcare (Basel, Switzerland) · 2026Review
- Navepegritide in children with achondroplasia.Translational pediatrics · 2026Article
- Achondroplasia management in the era of targeted therapies: a meta-analysis of C-type natriuretic peptide analogs.Journal of the Endocrine Society · 2026Review
- Real-world growth outcomes of vosoritide in children with achondroplasia: a single-center cohort study.Journal of the Endocrine Society · 2026Article
- C-Type Natriuretic Peptide Derivatives: Orthopaedic Implications in Achondroplasia.Journal of the Pediatric Orthopaedic Society of North America · 2026Review
- Effect of vosoritide on spine morphology in children with achondroplasia: 1-year results from a randomized phase 2 study.Journal of the Endocrine Society · 2026Article
- Development of Muscle Function in Children with Achondroplasia Under Vosoritide Treatment: A Retrospective Single-Centre Observational Study.Journal of musculoskeletal & neuronal interactions · 2026Observational
- The CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Clinical Outcomes in Untreated and Vosoritide-Treated Individuals.Hormone research in paediatrics · 2026Article
- Consensus statement on the management of Achondroplasia in the Middle East.Frontiers in endocrinology · 2026Article
- Functional Independence Assessment in Children and Adolescents with Achondroplasia: A Multicenter Cross-Sectional Study Using the WeeFIM Scale.Diagnostics (Basel, Switzerland) · 2025Article
- Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results from a Single Center in Portugal.Advances in therapy · 2025Article
- Review
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Context: Vosoritide is the first approved targeted therapy for achondroplasia (ACH) based on increased annualized growth velocity in clinical trials. The aim of our project was an assessment of the real-world setting and treatment with vosoritide. Design: This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide. Patients and Methods: Thirty-four patients with ACH (22 males; aged 2.8 to 15.3 years at treatment initiation) who received vosoritide treatment for at least 12 months at a specialized clinic for skeletal dysplasia in childhood were included in the analysis. Auxological measurements at baseline and after 12 months of therapy were converted into disease-specific (ACH) and general population [Centers for Disease Control and Prevention (CDC)] z-scores. Physical function assessed by a 6-minute walk test was converted into z-scores and compared to an unaffected reference cohort. Results: After 12 months of treatment, both ACH and CDC height z-scores showed significant increases, with mean changes (mean ± SD) of 0.52 ± 0.35 and 0.38 ± 0.44, respectively (both Conclusion: In a real-world setting, children with ACH showed significant improvements in growth and physical function after 12 months of treatment with vosoritide.
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