{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T00:55:13Z","timestamp":1793235313513,"version":"4.5.0"},"reference-count":17,"publisher":"Scientific Scholar","content-domain":{"domain":["ijmr.org.in"],"crossmark-restriction":true},"short-container-title":["Indian J Med Res"],"abstract":"<jats:sec>\n                    <jats:title>Background and objectives<\/jats:title>\n                    <jats:p>Achondroplasia, a rare autosomal dominant disorder caused by FGFR3 mutation, is treated with vosoritide, a C-type natriuretic peptide analogue. This review aimed to assess vosoritide\u2019s efficacy and safety to guide clinical practice and healthcare policy decisions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Advanced search was performed on three databases. The included articles underwent screening, data extraction, and quality evaluation, culminating in a qualitative synthesis. The protocol was registered on PROSPERO (CRD42024541795).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Out of 752 records screened, six were incorporated in this systematic review. The trials included daily injections of vosoritide (2.5-30 \u00b5g\/kg) in phase II study and at 15 \u00b5g\/kg in all other studies to patients with achondroplasia aged 3 months to 18 years. Safety assessments were done for all patients (n=156) having received vosoritide. Efficacy was assessed using annualised growth velocity and height Z score as the primary outcomes andserum collagen X-marker concentrations, bone age progression, and serum immunogenicity as secondary outcomes. Overall, the articles demonstrated good quality with minimal bias.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation and conclusions<\/jats:title>\n                    <jats:p>Vosoritide showed significant improvement in the annualised growth velocity, height z score and standing height in patients with achondroplasia aged 3 months to 18 years as compared to placebo. The safety assessment recorded adverse events in all patients (n=156) enrolled, usually mild (grade 1), self-limiting and limited to local injection site reactions. Studies with longer duration (till puberty), a large sample size and its effect on medical complications, are required to establish its effectiveness in the patients of achondroplasia.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.25259\/ijmr_2087_2025","type":"journal-article","created":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T16:26:10Z","timestamp":1772382370000},"page":"78-87","update-policy":"https:\/\/doi.org\/10.25259\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Efficacy and safety of Vosoritide in achondroplasia: A systematic review and meta-analysis"],"prefix":"10.25259","volume":"163","author":[{"given":"Monika","family":"Pahuja","sequence":"first","affiliation":[{"name":"Division of Discovery Research, Indian Council of Medical Research, New Delhi, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chander","family":"Prakash","sequence":"additional","affiliation":[{"name":"Division of Discovery Research, Indian Council of Medical Research, New Delhi, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankita","family":"Umrao","sequence":"additional","affiliation":[{"name":"Division of Discovery Research, Indian Council of Medical Research, New Delhi, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nabendu Sekhar","family":"Chatterjee","sequence":"additional","affiliation":[{"name":"Division of Discovery Research, Indian Council of Medical Research, New Delhi, India"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10894","published-online":{"date-parts":[[2026,2,28]]},"reference":[{"key":"10.25259\/IJMR_2087_2025\/ref-1","doi-asserted-by":"publisher","first-page":"1819","DOI":"10.1002\/ajmg.a.40356","article-title":"Development of body proportions in achondroplasia: Sitting height, leg length, arm span, and foot length","volume":"176","author":"Merker","year":"2018","journal-title":"Am J Med Genet A."},{"key":"10.25259\/IJMR_2087_2025\/ref-2","doi-asserted-by":"publisher","first-page":"23","DOI":"10.1210\/edrv.21.1.0387","article-title":"The molecular and genetic basis of fibroblast growth factor receptor 3 disorders: The achondroplasia family of skeletal dysplasias, Muenke craniosynostosis, and Crouzon syndrome with Acanthosis nigricans","volume":"21","author":"Vajo","year":"2000","journal-title":"Endocr Rev."},{"key":"10.25259\/IJMR_2087_2025\/ref-3","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1016\/j.ymgme.2017.10.002","article-title":"Clinical management of achondroplasia","volume":"122","author":"Hoover-Fong","year":"2017","journal-title":"Mol Genet Metab."},{"key":"10.25259\/IJMR_2087_2025\/ref-4","doi-asserted-by":"publisher","first-page":"705","DOI":"10.1136\/jmg.35.9.705","article-title":"Medical complications of achondroplasia: A multicentre patient review","volume":"35","author":"Hunter","year":"1998","journal-title":"J Med 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