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Accessed Mar 27, 2024."}],"container-title":["Advances in Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-024-03061-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12325-024-03061-y\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-024-03061-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,27]],"date-time":"2025-02-27T03:54:06Z","timestamp":1740628446000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12325-024-03061-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,2,5]]},"references-count":51,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,3]]}},"alternative-id":["3061"],"URL":"https:\/\/doi.org\/10.1007\/s12325-024-03061-y","relation":{},"ISSN":["0741-238X","1865-8652"],"issn-type":[{"value":"0741-238X","type":"print"},{"value":"1865-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,2,5]]},"assertion":[{"value":"31 May 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"30 October 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"5 February 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Julie Hoover-Fong has received honoraria from Ascendis, BioMarin, Innoskel, Medscape, QED Therapeutics, and Tyra for consulting, advisory board, and\/or speaking for educational purposes, and participated as an investigator in clinical trials funded by Alexion, BioMarin, QED Therapeutics and Therachon\/Pfizer.\u00a0Oliver Semler has received speaker honoraria and travel support from BioMarin. Bobbie Barron is an employee of and receives funding from the Institute for Clinical and Translational Research, which is funded by the National Center for Advancing Translational Sciences through the Clinical & Translational Science Awards Program, grant number UL1TR003098. Paulo Ferrez Collett-Solberg has received speaker honoraria and travel support from BioMarin and from producers of recombinant human growth hormone. Ellen Fung was previously a consultant to BioMarin, and now receives grant funding from BioMarin for a research study of vosoritide in patients with mucopolysaccaridosis. Melita Irving has received honoraria from BioMarin for speaking, for participating in advisory board meetings, and as a member of the vosoritide experts steering committee. Taichi Kitaoka has received honoraria for lectures from BioMarin. Celide Koerner has no conflicts of interest to report. Keita Okada has received honoraria for lectures and travel support from BioMarin. Katja Palm has received speaker honoraria and travel support from BioMarin, and travel support from producers of growth hormone. S\u00e9rgio B. Sousa has received honoraria from BioMarin, Kyowa Kirin, and Ascendis for speaking, for participating in advisory board meetings, and for travel support; and participated as an investigator in achondroplasia clinical trials funded by Ascendis and Therachon\/Pfizer. Klaus Mohnike has received honoraria from BioMarin, Novo-Nordisk and QED\/Biogridge for speaking, for participating in advisory board meetings, and as a member of steering committees.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"The pilot study briefly described in this commentary was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and was approved by the JHMI Institutional Review Board.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}