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Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.

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Title: Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.
Authors: Savarirayan R; Murdoch Children's Research Institute, Royal Children's Hospital Victoria, University of Melbourne, Parkville, Victoria, Australia. Electronic address: ravi.savarirayan@mcri.edu.au., Irving M; Guy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, United Kingdom., Wilcox WR; Emory University, Atlanta, GA., Bacino CA; Baylor College of Medicine, Houston, TX., Hoover-Fong JE; Johns Hopkins University School of Medicine, Baltimore, MD., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA., Polgreen LE; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA., Mohnike K; Otto-von-Guericke Universität, Universitätskinderklinik, Magdeburg, Germany., Prada CE; Division of Genetics, Genomics and Metabolism, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL., Kubota T; Osaka University, Osaka, Japan., Arundel P; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, United Kingdom., Leiva-Gea A; Instituto de Investigación Biomédica de Málaga-Plataforma Bionand, Málaga, Andalucía, Spain; Hospital Universitario Virgen de la Victoria, Málaga, Spain., Rowell R; BioMarin Pharmaceutical Inc., Novato, CA., Low A; BioMarin (United Kingdom) Limited, London, United Kingdom., Sabir I; BioMarin (United Kingdom) Limited, London, United Kingdom., Huntsman-Labed A; BioMarin (United Kingdom) Limited, London, United Kingdom., Day J; BioMarin (United Kingdom) Limited, London, United Kingdom.
Source: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2024 Dec; Vol. 26 (12), pp. 101274. Date of Electronic Publication: 2024 Sep 18.
Publication Type: Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 9815831 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1530-0366 (Electronic) Linking ISSN: 10983600 NLM ISO Abbreviation: Genet Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.
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  Data: <searchLink fieldCode="AU" term="%22Savarirayan+R%22">Savarirayan R</searchLink>; Murdoch Children's Research Institute, Royal Children's Hospital Victoria, University of Melbourne, Parkville, Victoria, Australia. Electronic address: ravi.savarirayan@mcri.edu.au.<br /><searchLink fieldCode="AU" term="%22Irving+M%22">Irving M</searchLink>; Guy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Wilcox+WR%22">Wilcox WR</searchLink>; Emory University, Atlanta, GA.<br /><searchLink fieldCode="AU" term="%22Bacino+CA%22">Bacino CA</searchLink>; Baylor College of Medicine, Houston, TX.<br /><searchLink fieldCode="AU" term="%22Hoover-Fong+JE%22">Hoover-Fong JE</searchLink>; Johns Hopkins University School of Medicine, Baltimore, MD.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, CA.<br /><searchLink fieldCode="AU" term="%22Polgreen+LE%22">Polgreen LE</searchLink>; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA.<br /><searchLink fieldCode="AU" term="%22Mohnike+K%22">Mohnike K</searchLink>; Otto-von-Guericke Universität, Universitätskinderklinik, Magdeburg, Germany.<br /><searchLink fieldCode="AU" term="%22Prada+CE%22">Prada CE</searchLink>; Division of Genetics, Genomics and Metabolism, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL.<br /><searchLink fieldCode="AU" term="%22Kubota+T%22">Kubota T</searchLink>; Osaka University, Osaka, Japan.<br /><searchLink fieldCode="AU" term="%22Arundel+P%22">Arundel P</searchLink>; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Leiva-Gea+A%22">Leiva-Gea A</searchLink>; Instituto de Investigación Biomédica de Málaga-Plataforma Bionand, Málaga, Andalucía, Spain; Hospital Universitario Virgen de la Victoria, Málaga, Spain.<br /><searchLink fieldCode="AU" term="%22Rowell+R%22">Rowell R</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA.<br /><searchLink fieldCode="AU" term="%22Low+A%22">Low A</searchLink>; BioMarin (United Kingdom) Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Sabir+I%22">Sabir I</searchLink>; BioMarin (United Kingdom) Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Huntsman-Labed+A%22">Huntsman-Labed A</searchLink>; BioMarin (United Kingdom) Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Day+J%22">Day J</searchLink>; BioMarin (United Kingdom) Limited, London, United Kingdom.
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  Data: <searchLink fieldCode="JN" term="%229815831%22">Genetics in medicine : official journal of the American College of Medical Genetics</searchLink> [Genet Med] 2024 Dec; Vol. 26 (12), pp. 101274. <i>Date of Electronic Publication: </i>2024 Sep 18.
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