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
Description
ISSN:2352-4650
DOI:10.1016/S2352-4642(23)00265-1