{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T10:01:33Z","timestamp":1780308093523,"version":"3.54.0"},"reference-count":24,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T00:00:00Z","timestamp":1773187200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T00:00:00Z","timestamp":1773187200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J of Med Genetics Pt A"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Vosoritide, a C\u2010type natriuretic peptide analogue that inhibits FGFR3 signaling, is approved for the treatment of achondroplasia (ACH) from birth in Japan, Australia, and the United States; however, data on neonatal use remain limited. We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date. Both patients tolerated treatment in the short term without serious adverse events during inpatient or outpatient monitoring. Despite early initiation, serial magnetic resonance imaging demonstrated progressive foramen magnum stenosis during infancy, and both patients required decompression surgery at 9 and 5\u2009months of age, respectively, consistent with the known natural history of ACH, with an uncomplicated postoperative course in both cases. Growth trajectories, assessed by growth velocity (GV), were generally above the ACH\u2010specific reference medians at comparable ages, while the characteristic slowing of GV during infancy was preserved. These observations are descriptive and hypothesis\u2010generating only and do not support changes in current clinical practice. Further data are required to clarify the potential benefits and limitations of initiating vosoritide therapy during the neonatal period.<\/jats:p>","DOI":"10.1002\/ajmg.a.70116","type":"journal-article","created":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T10:52:21Z","timestamp":1773226341000},"page":"1710-1715","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Early Neonatal Administration of Vosoritide in Achondroplasia: A Report of Two Cases"],"prefix":"10.1002","volume":"200","author":[{"given":"Shinichiro","family":"Sano","sequence":"first","affiliation":[{"name":"Department of Diabetes and Metabolism Shizuoka Children's Hospital  Shizuoka Japan"},{"name":"Department of Pediatrics Hamamatsu University School of Medicine  Hamamatsu Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuki","family":"Murai","sequence":"additional","affiliation":[{"name":"Department of Diabetes and Metabolism Shizuoka Children's Hospital  Shizuoka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2732-8722","authenticated-orcid":false,"given":"Kenji","family":"Shimizu","sequence":"additional","affiliation":[{"name":"Division of Genetics Shizuoka Children's Hospital  Shizuoka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasunori","family":"Nagai","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery Shizuoka Children's Hospital  Shizuoka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryuji","family":"Ishizaki","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery Shizuoka Children's Hospital  Shizuoka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshinobu","family":"Honda","sequence":"additional","affiliation":[{"name":"Department of Premature and Neonatal Medicine Iwaki City Medical Center  Fukushima Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,3,11]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.3171\/ped\u201007\/08\/152"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1210\/jcemcr\/luae144"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1136\/archdischild\u20102020\u2010319625"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.61120"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.61787"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/ped.70089"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.40528"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2021.115872"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469\u20108749.2012.04234.x"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010023\u201002795\u20102"},{"key":"e_1_2_13_12_1","first-page":"771","article-title":"Joint Recommendations on Sedation for Pediatric MRI Examinations","volume":"124","author":"Japan Pediatric Society, Japanese Society of Pediatric Anesthesiology, and Japanese Society of Pediatric Radiolog","year":"2020","journal-title":"Journal of the Japan Pediatric Society"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1297\/cpe.29.25"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn339"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.3171\/2018.9.Peds18410"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/dvdy.24479"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0022\u20103476(84)81092\u20106"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1093\/jbmr\/zjae173"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.63612"},{"key":"e_1_2_13_20_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41574\u2010024\u201001074\u20109"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41574\u2010021\u201000595\u2010x"},{"key":"e_1_2_13_22_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1813446"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0140\u20106736(20)31541\u20105"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/s2352\u20104642(23)00265\u20101"},{"key":"e_1_2_13_25_1","first-page":"1363","article-title":"Height Assessment in Children With Achondroplasia Based on a Nationwide Survey","volume":"60","author":"Tachibana K. 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