{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T21:36:48Z","timestamp":1740173808230,"version":"3.37.3"},"reference-count":34,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,11,28]],"date-time":"2024-11-28T00:00:00Z","timestamp":1732752000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.es","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Anales de Pediatr\u00eda (English Edition)"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1016\/j.anpede.2024.09.012","type":"journal-article","created":{"date-parts":[[2024,12,6]],"date-time":"2024-12-06T12:35:28Z","timestamp":1733488528000},"page":"401-410","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Expert consensus for the management of patients with achondroplasia in treatment with vosoritide"],"prefix":"10.1016","volume":"101","author":[{"given":"Ana-Coral","family":"Barreda-Bonis","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Josep Maria","family":"de Bergua Domingo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Enrique","family":"Gal\u00e1n-G\u00f3mez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Encarna","family":"Guill\u00e9n-Navarro","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Isabel","family":"Leiva-Gea","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Isolina","family":"Ria\u00f1o-Gal\u00e1n","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.anpede.2024.09.012_bib0005","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1038\/s41574-021-00595-x","article-title":"International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia","volume":"18","author":"Savarirayan","year":"2022","journal-title":"Nat Rev Endocrinol"},{"key":"10.1016\/j.anpede.2024.09.012_bib0010","doi-asserted-by":"crossref","first-page":"2297","DOI":"10.1002\/ajmg.a.61787","article-title":"Birth prevalence of achondroplasia: A systematic literature review and meta-analysis","volume":"182","author":"Foreman","year":"2020","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.anpede.2024.09.012_bib0015","unstructured":"Achondroplasia Synonym: FGFR3-Related Achondroplasia. GeneReviews\u00ae, 2023 [Accessed July 2024]. Available from: https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK1152\/GeneReviews."},{"key":"10.1016\/j.anpede.2024.09.012_bib0020","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s13023-018-0972-6","article-title":"Achondroplasia: a comprehensive clinical review","volume":"14","author":"Pauli","year":"2019","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.anpede.2024.09.012_bib0025","doi-asserted-by":"crossref","first-page":"2502","DOI":"10.1002\/ajmg.a.31919","article-title":"Mortality in achondroplasia study: a 42-year follow-up","volume":"143a","author":"Wynn","year":"2007","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.anpede.2024.09.012_bib0030","first-page":"27","article-title":"Estudio cl\u00ednico, epidemiol\u00f3gico, molecular y de calidad de vida de pacientes afectos de acondroplasia en Espa\u00f1a","volume":"2","author":"Gal\u00e1n G\u00f3mez","year":"2011","journal-title":"Rev Esp Endocrinol Pediatr"},{"key":"10.1016\/j.anpede.2024.09.012_bib0035","doi-asserted-by":"crossref","first-page":"2359","DOI":"10.1002\/ajmg.a.40528","article-title":"Multicenter study of mortality in achondroplasia","volume":"176","author":"Hashmi","year":"2018","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.anpede.2024.09.012_bib0040","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1186\/s13023-021-01971-6","article-title":"The first European consensus on principles of management for achondroplasia","volume":"16","author":"Cormier-Daire","year":"2021","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.anpede.2024.09.012_bib0045","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1007\/s00431-017-2923-y","article-title":"Final adult height in long-term growth hormone-treated achondroplasia patients","volume":"176","author":"Harada","year":"2017","journal-title":"Eur J Pediatr"},{"key":"10.1016\/j.anpede.2024.09.012_bib0050","doi-asserted-by":"crossref","first-page":"1665","DOI":"10.1007\/s00402-020-03360-3","article-title":"Staged upper and lower limb lengthening performing bilateral simultaneous surgery of the femur and tibia in achondroplastic patients","volume":"140","author":"Leiva-Gea","year":"2020","journal-title":"Arch Orthop Trauma Surg"},{"key":"10.1016\/j.anpede.2024.09.012_bib0055","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1016\/0092-8674(94)90302-6","article-title":"Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia","volume":"78","author":"Shiang","year":"1994","journal-title":"Cell"},{"key":"10.1016\/j.anpede.2024.09.012_bib0060","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0195876","article-title":"Early postnatal soluble FGFR3 therapy prevents the atypical development of obesity in achondroplasia","volume":"13","author":"Saint-Laurent","year":"2018","journal-title":"PLoS One"},{"key":"10.1016\/j.anpede.2024.09.012_bib0065","doi-asserted-by":"crossref","DOI":"10.1177\/00368504211003782","volume":"104","author":"Savarirayan","year":"2021","journal-title":"Sci Prog"},{"key":"10.1016\/j.anpede.2024.09.012_bib0070","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1007\/s11914-017-0347-2","article-title":"Current care and investigational therapies in achondroplasia","volume":"15","author":"Unger","year":"2017","journal-title":"Curr Osteoporos Rep"},{"key":"10.1016\/j.anpede.2024.09.012_bib0075","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1056\/NEJMoa1813446","article-title":"C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia","volume":"381","author":"Savarirayan","year":"2019","journal-title":"N Engl J Med"},{"key":"10.1016\/j.anpede.2024.09.012_bib0080","doi-asserted-by":"crossref","first-page":"684","DOI":"10.1016\/S0140-6736(20)31541-5","article-title":"Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial","volume":"396","author":"Savarirayan","year":"2020","journal-title":"Lancet"},{"key":"10.1016\/j.anpede.2024.09.012_bib0085","doi-asserted-by":"crossref","first-page":"2443","DOI":"10.1038\/s41436-021-01287-7","article-title":"Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study","volume":"23","author":"Savarirayan","year":"2021","journal-title":"Genet Med"},{"issue":"6","key":"10.1016\/j.anpede.2024.09.012_bib0090","doi-asserted-by":"crossref","DOI":"10.1542\/peds.2020-1010","article-title":"Health Supervision for People with Achondroplasia","volume":"145","author":"Hoover-Fong","year":"2020","journal-title":"Pediatrics"},{"key":"10.1016\/j.anpede.2024.09.012_bib0095","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1297\/cpe.29.25","article-title":"Clinical Practice Guidelines for Achondroplasia","volume":"29","author":"Kubota","year":"2020","journal-title":"Clin Pediatr Endocrinol"},{"key":"10.1016\/j.anpede.2024.09.012_bib0100","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1016\/j.anpedi.2022.10.004","article-title":"Achondroplasia: Update on diagnosis, follow-up and treatment","volume":"97","author":"Leiva-Gea","year":"2022","journal-title":"An Pediatr (Engl Ed)"},{"key":"10.1016\/j.anpede.2024.09.012_bib0105","doi-asserted-by":"crossref","first-page":"3639","DOI":"10.1007\/s12325-023-02549-3","article-title":"Burden and Treatment of Achondroplasia: A Systematic Literature Review","volume":"40","author":"Murton","year":"2023","journal-title":"Adv Ther"},{"key":"10.1016\/j.anpede.2024.09.012_bib0110","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1007\/s40618-023-02151-y","article-title":"The clinical management of children with achondroplasia in Italy: results of clinician and parent\/caregiver surveys","volume":"47","author":"Bedeschi","year":"2024","journal-title":"J Endocrinol Invest"},{"key":"10.1016\/j.anpede.2024.09.012_bib0115","doi-asserted-by":"crossref","first-page":"117","DOI":"10.2147\/TACG.S51485","article-title":"Optimal management of complications associated with achondroplasia","volume":"7","author":"Ireland","year":"2014","journal-title":"Appl Clin Genet"},{"key":"10.1016\/j.anpede.2024.09.012_bib0120","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1007\/s12325-023-02705-9","article-title":"Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice","volume":"41","author":"Semler","year":"2024","journal-title":"Adv Ther"},{"key":"10.1016\/j.anpede.2024.09.012_bib0125","unstructured":"European Medicines Agency. Annex I. Summary of product characteristics, 2023 [Accessed February 2024]. Available from: https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/voxzogo-epar-product-information_en.pdf."},{"key":"10.1016\/j.anpede.2024.09.012_bib0130","unstructured":"Informe de Posicionamiento Terap\u00e9utico de vosoritida (Voxzogo\u00ae) en el tratamiento de la acondroplasia en pacientes de 4 meses de edad y mayores cuyas ep\u00edfisis no se han cerrado, 2024 [Accessed July 2024]. Available from: https:\/\/www.aemps.gob.es\/medicamentosUsoHumano\/informesPublicos\/docs\/2023\/IPT-120-2023-Voxzogo.pdf."},{"key":"10.1016\/j.anpede.2024.09.012_bib0135","unstructured":"Puntos destacados de la reuni\u00f3n de la Comisi\u00f3n Interministerial de Precios de los Medicamentos 17 de julio de 2024, 2024 [Accessed July 2024]. Available from: https:\/\/www.sanidad.gob.es\/areas\/farmacia\/precios\/comisionInteministerial\/acuerdosNotasInformativas\/docs\/NOTAINFORMATIVACIPM_JULIO2024.pdf."},{"key":"10.1016\/j.anpede.2024.09.012_bib0140","doi-asserted-by":"crossref","first-page":"2457","DOI":"10.1007\/s12325-023-02496-z","article-title":"Parents\u2019 Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia","volume":"40","author":"NiMhurchadha","year":"2023","journal-title":"Adv Ther"},{"key":"10.1016\/j.anpede.2024.09.012_bib0145","article-title":"P193: Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: Update from phase 3 extension study","volume":"1","author":"Hoover-Fong","year":"2023","journal-title":"Genetics in Medicine Open"},{"key":"10.1016\/j.anpede.2024.09.012_bib0150","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1016\/S2352-4642(23)00265-1","article-title":"Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial","volume":"8","author":"Savarirayan","year":"2024","journal-title":"Lancet Child Adolesc Health"},{"key":"10.1016\/j.anpede.2024.09.012_bib0155","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1002\/ajmg.a.61974","article-title":"Clinical charts for surveillance of growth and body proportion development in achondroplasia and examples of their use","volume":"185","author":"Neumeyer","year":"2021","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.anpede.2024.09.012_bib0160","unstructured":"Achondroplasia Growth Charts. For growth monitoring of patients with achondroplasia, 2021 [Accessed August 2024]. Available from: https:\/\/www.achondroplasia-growthcharts.com\/."},{"key":"10.1016\/j.anpede.2024.09.012_bib0165","unstructured":"Informaci\u00f3n general sobre Voxzogo y sobre los motivos por los que se autoriza su uso en la UE, 2023 [Accessed March 2024]. Available from: https:\/\/www.ema.europa.eu\/es\/documents\/overview\/voxzogo-epar-medicine-overview_es.pdf."},{"key":"10.1016\/j.anpede.2024.09.012_bib0170","unstructured":"Protocole d\u2019utilisation th\u00e9rapeu-tique et de recueil de donn\u00e9es (PUT-RD). Acc\u00e8s pr\u00e9coce - VOXZOGO (vosoritide), 2023 [Accessed March 2024]. Available from: https:\/\/has-sante.fr\/plugins\/ModuleXitiKLEE\/types\/FileDocument\/doXiti.jsp?id=p_3304523."}],"container-title":["Anales de Pediatr\u00eda (English Edition)"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2341287924002862?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2341287924002862?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,12,13]],"date-time":"2024-12-13T16:51:55Z","timestamp":1734108715000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2341287924002862"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":34,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S2341287924002862"],"URL":"https:\/\/doi.org\/10.1016\/j.anpede.2024.09.012","relation":{},"ISSN":["2341-2879"],"issn-type":[{"type":"print","value":"2341-2879"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Expert consensus for the management of patients with achondroplasia in treatment with vosoritide","name":"articletitle","label":"Article Title"},{"value":"Anales de Pediatr\u00eda (English Edition)","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.anpede.2024.09.012","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.anpedi.2024.09.005","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 Published by Elsevier Espa\u00f1a, S.L.U. on behalf of Asociaci\u00f3n Espa\u00f1ola de Pediatr\u00eda.","name":"copyright","label":"Copyright"}]}}