{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T15:37:46Z","timestamp":1791041866008,"version":"4.1.0"},"reference-count":24,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2024,6,28]],"date-time":"2024-06-28T00:00:00Z","timestamp":1719532800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"BioMarin Pharmaceutical Australia Pty Ltd."}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Children"],"abstract":"<jats:p>Background: Achondroplasia, the most prevalent skeletal dysplasia, stems from a functional mutation in the fibroblast growth factor receptor 3 gene, leading to growth impairment. This condition presents multifaceted medical, functional and psychosocial challenges throughout childhood, adolescence and adulthood. Current management strategies aim to minimise medical complications, optimise functional capabilities and provide comprehensive supportive care. Vosoritide (trade name: VOXZOGO\u00ae, BioMarin Pharmaceuticals) is the first disease-modifying pharmaceutical treatment approved for the management of patients with achondroplasia and became available in Australia in May 2023. Methods: Standardised clinical guidelines for its optimal use are not yet widely available. To address this gap, a multidisciplinary Australian Vosoritide Working Group, comprising 12 experts with experience in achondroplasia management from across Australia, developed recommendations to guide the use of vosoritide in clinical practice. Results: The recommendations, which are expert opinions of the Australian Vosoritide Working Group, aim to (i) standardise the use of vosoritide across Australia, (ii) support the safe clinical rollout of vosoritide and (iii) support universal access. Conclusions: These recommendations have been developed for healthcare professionals and institutions that are engaged in using vosoritide in the management of achondroplasia and will be revised using a formal framework for clinical guideline development once more evidence is available.<\/jats:p>","DOI":"10.3390\/children11070789","type":"journal-article","created":{"date-parts":[[2024,6,28]],"date-time":"2024-06-28T06:51:53Z","timestamp":1719557513000},"page":"789","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Consensus Guidelines for the Use of Vosoritide in Children with Achondroplasia in Australia"],"prefix":"10.3390","volume":"11","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3961-0493","authenticated-orcid":false,"given":"Louise","family":"Tofts","sequence":"first","affiliation":[{"name":"Health and Human Sciences, Macquarie University, Macquarie Park, Sydney, NSW 2109, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Penny","family":"Ireland","sequence":"additional","affiliation":[{"name":"Queensland Children\u2019s Hospital, South Brisbane, QLD 4101, Australia"},{"name":"School of Health and Rehabilitation Sciences, University of Queensland, St Lucia, QLD 4072, Australia"},{"name":"Murdoch Children\u2019s Research Institute, Parkville, VIC 3052, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tracy","family":"Tate","sequence":"additional","affiliation":[{"name":"Kids Rehab, The Children\u2019s Hospital at Westmead, Sydney, NSW 2145, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Supriya","family":"Raj","sequence":"additional","affiliation":[{"name":"Murdoch Children\u2019s Research Institute, Parkville, VIC 3052, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Theresa","family":"Carroll","sequence":"additional","affiliation":[{"name":"Queensland Children\u2019s Hospital, South Brisbane, QLD 4101, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig F.","family":"Munns","sequence":"additional","affiliation":[{"name":"Queensland Children\u2019s Hospital, South Brisbane, QLD 4101, Australia"},{"name":"Child Health Research Centre, University of Queensland, South Brisbane, QLD 4101, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen","family":"Knipe","sequence":"additional","affiliation":[{"name":"The Newcastle Paediatric Clinic, New Lambton Heights, NSW 2305, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9729-6984","authenticated-orcid":false,"given":"Katherine","family":"Langdon","sequence":"additional","affiliation":[{"name":"Perth Children\u2019s Hospital, Perth, WA 6009, Australia"},{"name":"Telethon Kid\u2019s Institute, Perth, WA 6009, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lesley","family":"McGregor","sequence":"additional","affiliation":[{"name":"Women\u2019s and Children\u2019s Hospital, Adelaide, SA 5006, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4853-5269","authenticated-orcid":false,"given":"Fiona","family":"McKenzie","sequence":"additional","affiliation":[{"name":"Genetic Health WA, King Edward Memorial Hospital, Perth, WA 6008, Australia"},{"name":"School of Paediatrics and Child Health, University of Western Australia, Perth, WA 6009, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreas","family":"Zankl","sequence":"additional","affiliation":[{"name":"Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2050, Australia"},{"name":"Garvan Institute of Medical Research, Darlinghurst, NSW 2010, Australia"},{"name":"Department of Clinical Genetics, The Children\u2019s Hospital at Westmead, Sydney, NSW 2145, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ravi","family":"Savarirayan","sequence":"additional","affiliation":[{"name":"Murdoch Children\u2019s Research Institute, Parkville, VIC 3052, Australia"},{"name":"Murdoch Children\u2019s Research Institute, Royal Children\u2019s Hospital, University of Melbourne, Parkville, VIC 3052, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"166","DOI":"10.1186\/s13023-023-02755-w","article-title":"Real-world evidence in achondroplasia: Considerations for a standardized data set","volume":"18","author":"Alanay","year":"2023","journal-title":"Orphanet J. 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