Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.

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Title: Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.
Authors: Savarirayan R; Murdoch Children's Research Institute, Royal Children's Hospital, and University of Melbourne, Parkville, VIC, Australia. ravi.savarirayan@mcri.edu.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 WR; Department of Human Genetics, Emory University, Atlanta, GA, 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., Font RU; 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 Aydinlar University, School of Medicine, Istanbul, Turkey., Arundel P; Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, UK., Kotani Y; 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 Inc., Novato, CA, USA., Jayaram K; BioMarin Pharmaceutical Inc., Novato, CA, USA., Fisheleva E; BioMarin (U.K.) Limited, London, UK., Huntsman-Labed A; BioMarin (U.K.) Limited, London, UK., Day JRS; BioMarin (U.K.) Limited, London, UK.
Source: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2021 Dec; Vol. 23 (12), pp. 2443-2447. Date of Electronic Publication: 2021 Aug 02.
Publication Type: Clinical Trial, Phase III; 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
Description
ISSN:1530-0366
DOI:10.1038/s41436-021-01287-7