{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T04:03:41Z","timestamp":1792037021650,"version":"4.4.0"},"reference-count":31,"publisher":"The Endocrine Society","issue":"5","license":[{"start":{"date-parts":[[2025,3,8]],"date-time":"2025-03-08T00:00:00Z","timestamp":1741392000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,3,24]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Context<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Patients and Methods<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>After 12 months of treatment, both ACH and CDC height z-scores showed significant increases, with mean changes (mean \u00b1 SD) of 0.52 \u00b1 0.35 and 0.38 \u00b1 0.44, respectively (both P &amp;lt; .0001). The annualized growth velocity exceeded reference values for untreated children with ACH. No significant changes were observed in body mass index, upper to lower body segment ratio (sitting height\/height), or head circumference. The 6-minute walking distance improved, with z-scores increasing from \u22122.00 \u00b1 1.12 to \u22121.39 \u00b1 1.23 (P = .0215).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>In a real-world setting, children with ACH showed significant improvements in growth and physical function after 12 months of treatment with vosoritide.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1210\/jendso\/bvaf041","type":"journal-article","created":{"date-parts":[[2025,3,6]],"date-time":"2025-03-06T08:37:20Z","timestamp":1741250240000},"source":"Crossref","is-referenced-by-count":22,"title":["Real-world Outcome of Vosoritide Treatment in Children With Achondroplasia: A 12-month Retrospective Observational Study"],"prefix":"10.1210","volume":"9","author":[{"given":"Susanna","family":"Reincke","sequence":"first","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne , Cologne 50937 ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0029-7556","authenticated-orcid":false,"given":"Oliver","family":"Semler","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne , Cologne 50937 ,","place":["Germany"]},{"name":"Centre for Rare Skeletal Diseases in Childhood, Children's Hospital, University of Cologne , Cologne 50937 ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shino","family":"Jungh\u00e4nel-Welzing","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne , Cologne 50937 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,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5297-3662","authenticated-orcid":false,"given":"Heike","family":"Hoyer-Kuhn","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine, Faculty of Medicine and University Hospital Cologne, University of Cologne , Cologne 50937 ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2025,3,8]]},"reference":[{"issue":"6494","key":"2025040306511199100_bvaf041-B1","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1038\/371252a0","article-title":"Mutations in the gene encoding fibroblast growth factor receptor-3 in 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