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Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial.

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Title: Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 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., Wilcox WR; Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, USA., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Phillips J 3rd; Vanderbilt University Medical Center, Nashville, TN, USA., Polgreen LE; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA., Tofts L; Kids Rehab, The Children's Hospital at Westmead, Westmead, NSW, Australia., Ozono K; Osaka University Hospital, Osaka, Japan., Arundel P; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, UK., Irving M; Guy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, UK., Bacino CA; Baylor College of Medicine, Houston, TX, USA., Basel D; Medical College of Wisconsin, Milwaukee, WI, USA., Bober MB; Nemours/Alfred I du Pont Hospital for Children, Wilmington, DE, USA., Charrow J; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA., Mochizuki H; Saitama Children's Hospital, Saitama, Japan., Kotani Y; Tokushima University Hospital, Tokushima, Japan., Saal HM; Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Army C; BioMarin Pharmaceutical, Novato, CA, USA., Jeha G; BioMarin Pharmaceutical, Novato, CA, USA., Qi Y; BioMarin Pharmaceutical, Novato, CA, USA., Han L; 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: The Lancet. Child & adolescent health [Lancet Child Adolesc Health] 2024 Jan; Vol. 8 (1), pp. 40-50. Date of Electronic Publication: 2023 Nov 18.
Publication Type: Clinical Trial, Phase II; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Ltd Country of Publication: England NLM ID: 101712925 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-4650 (Electronic) Linking ISSN: 23524642 NLM ISO Abbreviation: Lancet Child Adolesc Health Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 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="%22Wilcox+WR%22">Wilcox WR</searchLink>; Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, USA.<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="%22Phillips+J+3rd%22">Phillips J 3rd</searchLink>; Vanderbilt University Medical Center, Nashville, TN, 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="%22Tofts+L%22">Tofts L</searchLink>; Kids Rehab, The Children's Hospital at Westmead, Westmead, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Ozono+K%22">Ozono K</searchLink>; Osaka University Hospital, Osaka, Japan.<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="%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="%22Bacino+CA%22">Bacino CA</searchLink>; Baylor College of Medicine, Houston, TX, USA.<br /><searchLink fieldCode="AU" term="%22Basel+D%22">Basel D</searchLink>; Medical College of Wisconsin, Milwaukee, WI, USA.<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="%22Charrow+J%22">Charrow J</searchLink>; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Mochizuki+H%22">Mochizuki H</searchLink>; Saitama Children's Hospital, Saitama, Japan.<br /><searchLink fieldCode="AU" term="%22Kotani+Y%22">Kotani Y</searchLink>; Tokushima University Hospital, Tokushima, Japan.<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="%22Army+C%22">Army C</searchLink>; BioMarin Pharmaceutical, Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Jeha+G%22">Jeha G</searchLink>; BioMarin Pharmaceutical, Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Qi+Y%22">Qi Y</searchLink>; BioMarin Pharmaceutical, Novato, CA, USA.<br /><searchLink fieldCode="AU" term="%22Han+L%22">Han L</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="%22101712925%22">The Lancet. Child & adolescent health</searchLink> [Lancet Child Adolesc Health] 2024 Jan; Vol. 8 (1), pp. 40-50. <i>Date of Electronic Publication: </i>2023 Nov 18.
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  Data: Clinical Trial, Phase II; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier+Ltd%22">Elsevier Ltd </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101712925 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2352-4650 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223524642%22">23524642 </searchLink><i>NLM ISO Abbreviation: </i>Lancet Child Adolesc Health <i>Subsets: </i>MEDLINE
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