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Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.

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Title: Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.
Authors: Savarirayan R; Murdoch Children's Research Institute, Royal Children's Hospital, and University of Melbourne, Parkville, VIC, Australia. Electronic address: ravi.savarirayan@vcgs.org.au., Tofts L; Kids Rehab, The Children's Hospital at Westmead, Westmead, NSW, Australia., Irving M; Guy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, UK., Wilcox W; Emory University, Atlanta, Georgia, USA., Bacino CA; Baylor College of Medicine, Houston, TX, USA., Hoover-Fong J; McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA., Ullot Font R; Hospital Sant Joan de Déu, Barcelona, Spain., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Rutsch F; Department of General Pediatrics, Muenster University Children's Hospital, Muenster, Germany., Bober MB; Nemours-Alfred I. du Pont Hospital for Children, Wilmington, DE, USA., Polgreen LE; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA., Ginebreda I; Hospital Universitario Quirón Dexeus, Barcelona, Spain., Mohnike K; Otto-von-Guericke-Universität, Magdeburg, Germany., Charrow J; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA., Hoernschemeyer D; University of Missouri-Columbia, Columbia, MO, USA., Ozono K; Osaka University Hospital, Osaka, Japan., Alanay Y; Acibadem Mehmet Ali Aydiniar University, School of Medicine, Istanbul, Turkey., Arundel P; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, UK., Kagami S; Tokushima University Hospital, Tokushima, Japan., Yasui N; Tokushima University Hospital, Tokushima, Japan., White KK; Seattle Children's Hospital, Seattle, WA, USA., Saal HM; Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Leiva-Gea A; Hospital Universitario Virgen de la Victoria, Málaga, Spain., Luna-González F; Hospital Universitario Virgen de la Victoria, Málaga, Spain., Mochizuki H; Saitama Children's Hospital, Saitama, Japan., Basel D; Medical College of Wisconsin, Milwaukee, WI, USA., Porco DM; BioMarin Pharmaceutical, Novato, CA, USA., Jayaram K; BioMarin Pharmaceutical, Novato, CA, USA., Fisheleva E; BioMarin (UK), London, UK., Huntsman-Labed A; BioMarin (UK), London, UK., Day J; BioMarin (UK), London, UK.
Source: Lancet (London, England) [Lancet] 2020 Sep 05; Vol. 396 (10252), pp. 684-692.
Publication Type: Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 2985213R Publication Model: Print Cited Medium: Internet ISSN: 1474-547X (Electronic) Linking ISSN: 01406736 NLM ISO Abbreviation: Lancet Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.
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  Data: <searchLink fieldCode="AU" term="%22Savarirayan+R%22">Savarirayan R</searchLink>; Murdoch Children's Research Institute, Royal Children's Hospital, and University of Melbourne, Parkville, VIC, Australia. Electronic address: ravi.savarirayan@vcgs.org.au.<br /><searchLink fieldCode="AU" term="%22Tofts+L%22">Tofts L</searchLink>; Kids Rehab, The Children's Hospital at Westmead, Westmead, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Irving+M%22">Irving M</searchLink>; Guy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, UK.<br /><searchLink fieldCode="AU" term="%22Wilcox+W%22">Wilcox W</searchLink>; Emory University, Atlanta, Georgia, USA.<br /><searchLink fieldCode="AU" term="%22Bacino+CA%22">Bacino CA</searchLink>; Baylor College of Medicine, Houston, TX, USA.<br /><searchLink fieldCode="AU" term="%22Hoover-Fong+J%22">Hoover-Fong J</searchLink>; McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Ullot+Font+R%22">Ullot Font R</searchLink>; Hospital Sant Joan de Déu, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.<br /><searchLink fieldCode="AU" term="%22Rutsch+F%22">Rutsch F</searchLink>; Department of General Pediatrics, Muenster University Children's Hospital, Muenster, Germany.<br /><searchLink fieldCode="AU" term="%22Bober+MB%22">Bober MB</searchLink>; Nemours-Alfred I. du Pont Hospital for Children, Wilmington, DE, USA.<br /><searchLink fieldCode="AU" term="%22Polgreen+LE%22">Polgreen LE</searchLink>; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA.<br /><searchLink fieldCode="AU" term="%22Ginebreda+I%22">Ginebreda I</searchLink>; Hospital Universitario Quirón Dexeus, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Mohnike+K%22">Mohnike K</searchLink>; Otto-von-Guericke-Universität, Magdeburg, Germany.<br /><searchLink fieldCode="AU" term="%22Charrow+J%22">Charrow J</searchLink>; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Hoernschemeyer+D%22">Hoernschemeyer D</searchLink>; University of Missouri-Columbia, Columbia, MO, USA.<br /><searchLink fieldCode="AU" term="%22Ozono+K%22">Ozono K</searchLink>; Osaka University Hospital, Osaka, Japan.<br /><searchLink fieldCode="AU" term="%22Alanay+Y%22">Alanay Y</searchLink>; Acibadem Mehmet Ali Aydiniar University, School of Medicine, Istanbul, Turkey.<br /><searchLink fieldCode="AU" term="%22Arundel+P%22">Arundel P</searchLink>; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, UK.<br /><searchLink fieldCode="AU" term="%22Kagami+S%22">Kagami S</searchLink>; Tokushima University Hospital, Tokushima, Japan.<br /><searchLink fieldCode="AU" term="%22Yasui+N%22">Yasui N</searchLink>; Tokushima University Hospital, Tokushima, Japan.<br /><searchLink fieldCode="AU" term="%22White+KK%22">White KK</searchLink>; Seattle Children's Hospital, Seattle, WA, USA.<br /><searchLink fieldCode="AU" term="%22Saal+HM%22">Saal HM</searchLink>; Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Leiva-Gea+A%22">Leiva-Gea A</searchLink>; Hospital Universitario Virgen de la Victoria, Málaga, Spain.<br /><searchLink fieldCode="AU" term="%22Luna-González+F%22">Luna-González F</searchLink>; Hospital Universitario Virgen de la Victoria, Málaga, Spain.<br /><searchLink fieldCode="AU" term="%22Mochizuki+H%22">Mochizuki H</searchLink>; Saitama Children's Hospital, Saitama, Japan.<br /><searchLink fieldCode="AU" term="%22Basel+D%22">Basel D</searchLink>; Medical College of Wisconsin, Milwaukee, WI, USA.<br /><searchLink fieldCode="AU" term="%22Porco+DM%22">Porco DM</searchLink>; BioMarin Pharmaceutical, Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Jayaram+K%22">Jayaram K</searchLink>; BioMarin Pharmaceutical, Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Fisheleva+E%22">Fisheleva E</searchLink>; BioMarin (UK), London, UK.<br /><searchLink fieldCode="AU" term="%22Huntsman-Labed+A%22">Huntsman-Labed A</searchLink>; BioMarin (UK), London, UK.<br /><searchLink fieldCode="AU" term="%22Day+J%22">Day J</searchLink>; BioMarin (UK), London, UK.
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  Data: <searchLink fieldCode="JN" term="%222985213R%22">Lancet (London, England)</searchLink> [Lancet] 2020 Sep 05; Vol. 396 (10252), pp. 684-692.
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  Data: Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
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