{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T16:43:34Z","timestamp":1789663414354,"version":"4.0.1"},"reference-count":41,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2023,10,26]],"date-time":"2023-10-26T00:00:00Z","timestamp":1698278400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2023,10,26]],"date-time":"2023-10-26T00:00:00Z","timestamp":1698278400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/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"}]},{"DOI":"10.13039\/501100012681","name":"Universit\u00e4tsklinikum K\u00f6ln","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100012681","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Adv Ther"],"published-print":{"date-parts":[[2024,1]]},"DOI":"10.1007\/s12325-023-02705-9","type":"journal-article","created":{"date-parts":[[2023,10,26]],"date-time":"2023-10-26T05:02:12Z","timestamp":1698296532000},"page":"198-214","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":32,"title":["Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice"],"prefix":"10.1007","volume":"41","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0029-7556","authenticated-orcid":false,"given":"Oliver","family":"Semler","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Val\u00e9rie","family":"Cormier-Daire","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ekkehart","family":"Lausch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6178-1264","authenticated-orcid":false,"given":"Michael B.","family":"Bober","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9221-4879","authenticated-orcid":false,"given":"Ricki","family":"Carroll","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3921-1334","authenticated-orcid":false,"given":"S\u00e9rgio B.","family":"Sousa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Deyle","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maha","family":"Faden","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriele","family":"Hartmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron J.","family":"Huser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6684-4909","authenticated-orcid":false,"given":"Janet M.","family":"Legare","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klaus","family":"Mohnike","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9516-7863","authenticated-orcid":false,"given":"Tilman R.","family":"Rohrer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank","family":"Rutsch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2588-9657","authenticated-orcid":false,"given":"Pamela","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4147-2800","authenticated-orcid":false,"given":"Andre M.","family":"Travessa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Verardo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klane K.","family":"White","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William R.","family":"Wilcox","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1242-5626","authenticated-orcid":false,"given":"Julie","family":"Hoover-Fong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,10,26]]},"reference":[{"key":"2705_CR1","doi-asserted-by":"publisher","first-page":"1791","DOI":"10.1002\/ajmg.a.61289","volume":"179","author":"A Coi","year":"2019","unstructured":"Coi A, Santoro M, Garne E, et al. Epidemiology of achondroplasia: a population-based study in Europe. Am J Med Genet A. 2019;179:1791\u20138.","journal-title":"Am J Med Genet A"},{"key":"2705_CR2","doi-asserted-by":"publisher","first-page":"2297","DOI":"10.1002\/ajmg.a.61787","volume":"182A","author":"PK Foreman","year":"2020","unstructured":"Foreman PK, van Kessel F, van Hoorn R, van den Bosch J, Shediac R, Landis S. Birth prevalence of achondroplasia: a systematic literature review and meta-analysis. Am J Med Genet A. 2020;182A:2297\u2013316.","journal-title":"Am J Med Genet A"},{"key":"2705_CR3","doi-asserted-by":"publisher","first-page":"252","DOI":"10.1038\/371252a0","volume":"371","author":"F Rousseau","year":"1994","unstructured":"Rousseau F, Bonaventure J, Legai-Mallet L, et al. Mutations in the gene encoding fibroblast growth factor receptor-3 in achondroplasia. Nature. 1994;371:252\u20134.","journal-title":"Nature"},{"key":"2705_CR4","doi-asserted-by":"publisher","first-page":"335","DOI":"10.1016\/0092-8674(94)90302-6","volume":"78","author":"R Shiang","year":"1994","unstructured":"Shiang R, Thompson LM, Zhu YZ, et al. Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia. Cell. 1994;78:335\u201342.","journal-title":"Cell"},{"key":"2705_CR5","doi-asserted-by":"publisher","first-page":"162","DOI":"10.1016\/S0140-6736(07)61090-3","volume":"370","author":"WA Horton","year":"2007","unstructured":"Horton WA, Hall JG, Hecht JT. Achondroplasia. Lancet. 2007;370:162\u201372.","journal-title":"Lancet"},{"key":"2705_CR6","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2021.115872","volume":"146","author":"J Hoover-Fong","year":"2021","unstructured":"Hoover-Fong J, Cheung MS, Fano V, et al. Lifetime impact of achondroplasia: current evidence and perspectives on the natural history. Bone. 2021;146:115872.","journal-title":"Bone"},{"key":"2705_CR7","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1136\/adc.2010.189092","volume":"97","author":"MJ Wright","year":"2012","unstructured":"Wright MJ, Irving M. Clinical management of achondroplasia. Arch Dis Child. 2012;97:129\u201334.","journal-title":"Arch Dis Child"},{"key":"2705_CR8","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13023-018-0972-6","volume":"14","author":"RM Pauli","year":"2019","unstructured":"Pauli RM. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis. 2019;14:1.","journal-title":"Orphanet J Rare Dis"},{"key":"2705_CR9","doi-asserted-by":"publisher","first-page":"56","DOI":"10.1186\/s13023-023-02652-2","volume":"18","author":"M Maghnie","year":"2023","unstructured":"Maghnie M, Semler O, Guillen-Navarro E, et al. Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study. Orphanet J Rare Dis. 2023;18:56.","journal-title":"Orphanet J Rare Dis"},{"key":"2705_CR10","doi-asserted-by":"publisher","first-page":"2444","DOI":"10.1016\/j.gim.2022.08.015","volume":"24","author":"R Savarirayan","year":"2022","unstructured":"Savarirayan R, Irving M, Harmatz P, et al. Growth parameters in children with achondroplasia: a 7-year, prospective, multinational, observational study. Genet Med. 2022;24:2444\u201352.","journal-title":"Genet Med"},{"key":"2705_CR11","doi-asserted-by":"publisher","first-page":"435","DOI":"10.1016\/S0022-3476(78)81152-4","volume":"93","author":"WA Horton","year":"1978","unstructured":"Horton WA, Rotter JI, Rimoin DL, Scott CI, Hall JG. Standard growth curves for achondroplasia. J Pediatr. 1978;93:435\u20138.","journal-title":"J Pediatr"},{"key":"2705_CR12","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1002\/ajmg.a.38853","volume":"176","author":"A Merker","year":"2018","unstructured":"Merker A, Neumeyer L, Hertel NT, et al. Growth in achondroplasia: development of height, weight, head circumference, and body mass index in a European cohort. Am J Med Genet A. 2018;176:1723\u201334.","journal-title":"Am J Med Genet A"},{"key":"2705_CR13","doi-asserted-by":"publisher","first-page":"1818","DOI":"10.1002\/ajmg.a.40356","volume":"176","author":"A Merker","year":"2018","unstructured":"Merker A, Neumeyer L, Hertal NT, et al. Development of body proportions in achondroplasia: sitting height, leg length, arm span, and foot length. Am J Med Genet A. 2018;176:1818\u201329.","journal-title":"Am J Med Genet A"},{"key":"2705_CR14","doi-asserted-by":"publisher","first-page":"522","DOI":"10.1186\/s13023-021-02141-4","volume":"16","author":"JE Hoover-Fong","year":"2021","unstructured":"Hoover-Fong JE, Schulze KJ, Alade AY, et al. Growth in achondroplasia including stature, weight, weight-for-height and head circumference from CLARITY: achondroplasia natural history study\u2014a multi-center retrospective cohort study of achondroplasia in the US. Orphanet J Rare Dis. 2021;16:522.","journal-title":"Orphanet J Rare Dis"},{"key":"2705_CR15","doi-asserted-by":"publisher","first-page":"e20201010","DOI":"10.1542\/peds.2020-1010","volume":"145","author":"J Hoover-Fong","year":"2020","unstructured":"Hoover-Fong J, Scott CI, Jones MC, AAP Committee on Genetics. Health supervision for people with achondroplasia. Pediatrics. 2020;145:e20201010.","journal-title":"Pediatrics."},{"key":"2705_CR16","doi-asserted-by":"publisher","first-page":"237","DOI":"10.1111\/cge.12045","volume":"84","author":"Y Alade","year":"2013","unstructured":"Alade Y, Tunkel D, Schulze K, et al. Cross-sectional assessment of pain and physical function in skeletal dysplasia patients. Clin Genet. 2013;84:237\u201343.","journal-title":"Clin Genet"},{"key":"2705_CR17","doi-asserted-by":"publisher","first-page":"6166","DOI":"10.1080\/09638288.2021.1963853","volume":"44","author":"C Constantinides","year":"2022","unstructured":"Constantinides C, Landis SH, Jarrett J, Quinn J, Ireland PJ. Quality of life, physical functioning, and psychosocial function among patients with achondroplasia: a targeted literature review. Disabil Rehabil. 2022;44:6166\u201378.","journal-title":"Disabil Rehabil"},{"key":"2705_CR18","doi-asserted-by":"publisher","first-page":"S127","DOI":"10.1016\/S1096-7192(21)00280-8","volume":"132","author":"M Maghnie","year":"2021","unstructured":"Maghnie M, Semler O, Guillen-Navarro E, et al. Health-related quality of life (HRQoL) in achondroplasia: findings from a multinational, observational study. Mol Genet Metabol. 2021;132:S127\u20138.","journal-title":"Mol Genet Metabol"},{"issue":"1","key":"2705_CR19","doi-asserted-by":"publisher","first-page":"139","DOI":"10.1186\/s13023-023-02738-x","volume":"18","author":"NJ Nahm","year":"2023","unstructured":"Nahm NJ, Mackenzie WS, Mackenzie WG, et al. Achondroplasia natural history study (CLARITY): 60-year experience in orthopedic surgery from four skeletal dysplasia centers. Orphanet J Rare Dis. 2023;18(1):139.","journal-title":"Orphanet J Rare Dis"},{"key":"2705_CR20","doi-asserted-by":"publisher","first-page":"1665","DOI":"10.1007\/s00402-020-03360-3","volume":"140","author":"A Leiva-Gea","year":"2020","unstructured":"Leiva-Gea A, Delgado-Rufino FB, Queipo-de-Llano A, Mariscal-Lara J, Lombardo-Torre M, Luna-Gonz\u00e1lez F. Staged upper and lower limb lengthening performing bilateral simultaneous surgery of the femur and tibia in achondroplastic patients. Arch Orthop Trauma Surg. 2020;140:1665\u201376.","journal-title":"Arch Orthop Trauma Surg"},{"key":"2705_CR21","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1186\/s10195-019-0541-3","volume":"21","author":"GA Hosny","year":"2020","unstructured":"Hosny GA. Limb lengthening history, evolution, complications and current concepts. J Orthop Traumatol. 2020;21:3.","journal-title":"J Orthop Traumatol"},{"issue":"Suppl 1","key":"2705_CR22","first-page":"123","volume":"16","author":"T Yorifuji","year":"2018","unstructured":"Yorifuji T, Higuchi S, Kawakita R. Growth hormone treatment for achondroplasia. Pediatr Endocrinol Rev. 2018;16(Suppl 1):123\u20138.","journal-title":"Pediatr Endocrinol Rev"},{"key":"2705_CR23","doi-asserted-by":"publisher","first-page":"873","DOI":"10.1007\/s00431-017-2923-y","volume":"176","author":"D Harada","year":"2017","unstructured":"Harada D, Namba N, Hanioka Y, et al. Final adult height in long-term growth hormone-treated achondroplasia patients. Eur J Pediatr. 2017;176:873\u20139.","journal-title":"Eur J Pediatr"},{"key":"2705_CR24","doi-asserted-by":"publisher","first-page":"1108","DOI":"10.1016\/j.ajhg.2012.10.014","volume":"91","author":"F Lorget","year":"2012","unstructured":"Lorget F, Kaci N, Peng J, et al. Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. Am J Hum Genet. 2012;91:1108\u201314.","journal-title":"Am J Hum Genet"},{"key":"2705_CR25","doi-asserted-by":"publisher","first-page":"25","DOI":"10.1056\/NEJMoa1813446","volume":"381","author":"R Savarirayan","year":"2019","unstructured":"Savarirayan R, Irving M, Bacino CA, et al. C-type natriuretic peptide analogue therapy in children with achondroplasia. N Eng J Med. 2019;381:25\u201335.","journal-title":"N Eng J Med"},{"key":"2705_CR26","doi-asserted-by":"publisher","first-page":"684","DOI":"10.1016\/S0140-6736(20)31541-5","volume":"396","author":"R Savarirayan","year":"2020","unstructured":"Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase\u00a03, placebo-controlled, multicentre trial. Lancet. 2020;396:684\u201392.","journal-title":"Lancet"},{"issue":"1","key":"2705_CR27","first-page":"100222","volume":"1","author":"J Hoover-Fong","year":"2023","unstructured":"Hoover-Fong J, Savarirayan R, Tofts L, et al. Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 35 years: update from phase\u00a03 extension study. Genet Med. 2023;1(1):100222.","journal-title":"Genet Med"},{"issue":"Suppl 2","key":"2705_CR28","first-page":"308","volume":"95","author":"V Cormier-Daire","year":"2022","unstructured":"Cormier-Daire V, Cohen S, Edouard T, et al. Real-world experience with vosoritide for achondroplasia: interim findings from an early access programme in France. Horm Res Paediatr. 2022;95(Suppl 2):308.","journal-title":"Horm Res Paediatr"},{"key":"2705_CR29","unstructured":"Cormier-Daire V, Edouard T, Isidor B, et al. Real-world safety and effectiveness of vosoritide: Results from an early-access program in France. Presented at the 2023 ESPE European Society for Paediatric Endocrinology, 21\u201323 September 2023, The Hague, Netherlands."},{"issue":"Suppl 2","key":"2705_CR30","first-page":"151","volume":"95","author":"K Palm","year":"2022","unstructured":"Palm K, Bechthold-Dalla Pozza S, Gausche R, et al. Real-world data in children with achondroplasia after licensing of vosoritide. Horm Res Peadiatr. 2022;95(Suppl 2):151.","journal-title":"Horm Res Peadiatr"},{"key":"2705_CR31","unstructured":"Kunkel P, Al Halak M, Bechthold-Dalla Pozza S, et al. Multidisciplinary approach in achondroplasia \u2013 real world experience after drug approval of vosoritide. Presented at the 2023 ESPE European Society for Paediatric Endocrinology, 21\u201323 September 2023, The Hague, Netherlands."},{"key":"2705_CR32","unstructured":"Voxzogo Summary of Product Characteristics 2021. https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/voxzogo-epar-product-information_en.pdf. Accessed 05 May 2023"},{"key":"2705_CR33","unstructured":"Voxzogo US Prescribing Information (2021, November). https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2021\/214938s000lbl.pdf Accessed 05 May 2023."},{"key":"2705_CR34","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1038\/s41574-021-00595-x","volume":"18","author":"R Savarirayan","year":"2022","unstructured":"Savarirayan R, Ireland P, Irving M, et al. International consensus statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022;18:173\u201389.","journal-title":"Nat Rev Endocrinol"},{"key":"2705_CR35","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1186\/s13023-021-01971-6","volume":"16","author":"V Cormier-Daire","year":"2021","unstructured":"Cormier-Daire V, AlSayed M, Ben-Omran T, et al. The first European consensus on principles of management for achondroplasia. Orphanet J Rare Dis. 2021;16:333.","journal-title":"Orphanet J Rare Dis"},{"key":"2705_CR36","unstructured":"National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Care Services; Committee on Health Care Utilization and Adults with Disabilities. Health-Care Utilization as a Proxy in Disability Determination. Washington (DC): National Academies Press (US); 2018."},{"key":"2705_CR37","doi-asserted-by":"publisher","first-page":"e20161485","DOI":"10.1542\/peds.2016-1485","volume":"138","author":"AL Katz","year":"2016","unstructured":"Katz AL, Webb SA, AAP Committee on Bioethics. Informed consent in decision-making in pediatric practice. Pediatrics. 2016;138:e20161485.","journal-title":"Pediatrics"},{"key":"2705_CR38","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-023-02496-z","author":"S NiMhurchadha","year":"2023","unstructured":"NiMhurchadha S, Butler K, Argent R, et al. Parents\u2019 experience of administering vosoritide: a daily injectable for children with achondroplasia. Adv Ther. 2023. https:\/\/doi.org\/10.1007\/s12325-023-02496-z.","journal-title":"Adv Ther"},{"key":"2705_CR39","unstructured":"Protocole d\u2019utilisation th\u00e9rapeutique et de recueil de donn\u00e9es (PUT-RD). Acc\u00e8s pr\u00e9coce\u2014VOXZOGO (vosoritide). https:\/\/has-sante.fr\/plugins\/ModuleXitiKLEE\/types\/FileDocument\/doXiti.jsp?id=p_3304523 Accessed 17 May 2023."},{"key":"2705_CR40","unstructured":"Voxzogo exclusion criteria: an extension study to evaluate the efficacy and safety of BMN 111 in children with achondroplasia, 2018. https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT03424018?term=302&cond=BMN111&draw=2&rank=1 Accessed 05 May 2023."},{"key":"2705_CR41","doi-asserted-by":"publisher","DOI":"10.1097\/00000441-195909000-00030","volume-title":"Radiographic atlas of skeletal development of the hand and wrist","author":"W Greulich","year":"1959","unstructured":"Greulich W, Pyle I. Radiographic atlas of skeletal development of the hand and wrist. London: Stanford University Press; 1959."}],"container-title":["Advances in Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-023-02705-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12325-023-02705-9\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-023-02705-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,18]],"date-time":"2024-01-18T17:06:15Z","timestamp":1705597575000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12325-023-02705-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,26]]},"references-count":41,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,1]]}},"alternative-id":["2705"],"URL":"https:\/\/doi.org\/10.1007\/s12325-023-02705-9","relation":{},"ISSN":["0741-238X","1865-8652"],"issn-type":[{"value":"0741-238X","type":"print"},{"value":"1865-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,10,26]]},"assertion":[{"value":"1 June 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"3 October 2023","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"26 October 2023","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"In addition to the individual conflict of interest outlined below, all authors participated in the expert group meetings, sponsored and organized by BioMarin Pharmaceutical Inc, on which this manuscript is based. Oliver Semler: has received grants or contracts from Kyowa Kirin, payment or honoraria for lectures\/presentations from BioMarin and Kyowa Kirin, and support for attending meetings and\/or travel from Pfizer. Val\u00e9rie Cormier-Daire: has received payment or honoraria for lectures\/presentations from BioMarin and Ipsen, and participated in Advisory Board meetings for BioMarin, QED-Propel and Mereo. Ekkehart Lausch: has received grants or contracts from DFG (SFB1453), H2020, consulting fees, support for attending meetings and\/or travel, and has participated in Advisory Board meetings for BioMarin. Michael B. Bober: has received grants or contracts from BioMarin, Ascendis, QED and Pfizer (all Institutional), consulting fees from BioMarin and Tyra (Institutional), Ascendis and Bridge Bio, payment or honoraria for lectures\/presentations from NovoNordisk and Tyra, has participated in Advisory Board meetings for BioMarin, and has a leadership role for the Skeletal Dysplasia Management Consortium. Ricki Carroll: has received grants or contracts from BioMarin, QED, Ascendis and Pfizer (all Institutional), and consulting fees from Guidepoint. S\u00e9rgio B. Sousa: has received payment or honoraria for lectures\/presentations, support for attending meetings and\/or travel, and has participated in Advisory Board meetings for BioMarin, and has a leadership role for the European Achondroplasia Forum and ERN-BOND. David Deyle: has received grants or contracts from Castle Creek. Maha Faden: has received support for attending meetings and\/or travel, and has participated in Advisory Board meetings for BioMarin. Gabriele Hartmann: has received consulting fees, support for attending meetings and\/or travel, and has participated in Advisory Board meetings for BioMarin. Aaron J. Huser: has received payment or honoraria for speaker bureau, has participated in Advisory Board meetings for BioMarin, and holds a leadership role for the MHE Research Foundation. Janet M. Legare: has received grants or contracts for clinical trials from Ascendis Pharma and Bridge Bio, and for a natural history study from BioMarin, and payment or honoraria for lectures\/presentations from BioMarin. Klaus Mohnike: has received grants or contracts for clinical trials, payment or honoraria for lectures\/presentations, support for attending meetings and\/or travel and has participated in Advisory Board meetings for BioMarin. Tilman R. Rohrer: has received grants or contracts, consulting fees, payment or honoraria for lectures\/presentations and support for attending meetings and\/or travel from BioMarin and NovoNordisk. Frank Rutsch: has received grants or contracts (Institutional), consulting fees, payment or honoraria for lectures\/presentations, support for attending meetings and\/or travel, and has participated in Advisory Board meetings for BioMarin. Pamela Smith: has received payment or honoraria for lectures\/presentations, and support for attending meetings and\/or travel from BioMarin. Andre M. Travessa: has received consulting fees, support for attending meetings and\/or travel, and participated in Advisory Board meetings. Angela Verardo: Has received payment or honoraria for speaker bureau from BioMarin. Klane K. White: Has received grants or contracts from BioMarin, Ascendis, Ultragenyx and Pfizer, royalties or licenses for UptoDate.com, payment or honoraria for lectures\/presentations from BioMarin and has a leadership role for Little People of America, National MPS Society, Pediatric Orthopedic Society of North America, Scoliosis Research Society, Camp Korey, and the Skeletal Dysplasia Management Consortium. William R. Wilcox: has received grants or contracts (Institutional), and has participated in Advisory Board meetings for BioMarin. Julie Hoover-Fong: has received grants or contracts for clinical trials from BioMarin, QED and Pfizer, consulting fees from BioMarin, QED, NovoNordisk and Innoskel, payment or honoraria for lectures\/presentations from Medscape and BioMarin, has participated in Advisory Board meetings for MCDS\u2014Therapy Clinical Trial (unpaid), BioMarin and QED, and is an unpaid member of the Skeletal Dysplasia Management Consortium and of the Medical Advisory Board of the Little People of America.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"This article is based on previously conducted studies and does not contain any new studies with human participants or animals performed by any of the authors.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}