{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T07:10:46Z","timestamp":1780384246212,"version":"3.54.1"},"reference-count":21,"publisher":"The Endocrine Society","issue":"6","license":[{"start":{"date-parts":[[2026,3,28]],"date-time":"2026-03-28T00:00:00Z","timestamp":1774656000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Medison Pharma Ltd"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,5,6]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Context<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To evaluate real-world growth outcomes of vosoritide therapy in children with achondroplasia, including those with prior surgical interventions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>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. \u0394 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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Mean height z-scores improved from \u22120.62 \u00b1 1.09 to \u22120.24 \u00b1 1.20 (P &amp;lt; .001). Mean arm span z-score increased from \u22121.28 \u00b1 0.93 to -0.96 \u00b1 0.91 (P = .007). Sitting height z-scores showed a non-significant trend toward improvement; BMI z-scores remained stable. Mean \u0394 height and \u0394 arm span z-score gains were +0.38 \u00b1 0.45 and +0.32 \u00b1 0.48, respectively. A subgroup analysis of 8 patients with vs 17 without prior limb-lengthening surgery showed no significant differences. Regression analyses adjusted for sex, age, BMI z-scores, and surgical history were likely underpowered to detect meaningful predictors of response. There were no serious treatment-related adverse events.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1210\/jendso\/bvag072","type":"journal-article","created":{"date-parts":[[2026,3,26]],"date-time":"2026-03-26T12:45:10Z","timestamp":1774529110000},"source":"Crossref","is-referenced-by-count":0,"title":["Real-world growth outcomes of vosoritide in children with achondroplasia: a single-center cohort study"],"prefix":"10.1210","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0846-733X","authenticated-orcid":false,"given":"Ravit","family":"Regev","sequence":"first","affiliation":[{"name":"Institute of Pediatric Endocrinology, Diabetes and Metabolism, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv 6423906, Israel, affiliated to The Gray Faculty of Medical & Health Sciences, Tel Aviv University , Tel Aviv, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yarden","family":"Waksman","sequence":"additional","affiliation":[{"name":"Institute of Pediatric Endocrinology, Diabetes and Metabolism, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv 6423906, Israel, affiliated to The Gray Faculty of Medical & Health Sciences, Tel Aviv University , Tel Aviv, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-0083-9443","authenticated-orcid":false,"given":"Eyal","family":"Cohen-Sela","sequence":"additional","affiliation":[{"name":"Institute of Pediatric Endocrinology, Diabetes and Metabolism, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv 6423906, Israel, affiliated to The Gray Faculty of Medical & Health Sciences, Tel Aviv University , Tel Aviv, 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