Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study.

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Abstract

This study was undertaken to collect baseline growth parameters in children with achondroplasia who might enroll in interventional trials of vosoritide, and to establish a historical control.

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Journal

Journal ID (iso-abbrev): Genet Med

Title: Genetics in medicine : official journal of the American College of Medical Genetics

Publisher: Elsevier BV

ISSN (Electronic): 1530-0366

ISSN (Print): 1098-3600

Publication date (Electronic): Dec 2022

Volume: 24

Issue: 12

Affiliations

[1 ] Murdoch Children's Research Institute, Royal Children's Hospital and University of Melbourne, Parkville, Victoria, Australia. Electronic address: ravi.savarirayan@vcgs.org.au.

[2 ] Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation Trust, London, United Kingdom.

[3 ] UCSF Benioff Children's Hospital Oakland, Oakland, CA.

[4 ] Hospital Universitario Virgen de la Victoria, Málaga, Spain.

[5 ] Department of Human Genetics, Emory University School of Medicine, Emory University, Atlanta, GA.

[6 ] Vanderbilt University Medical Center, Nashville, TN.

[7 ] Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX.

[8 ] Kids Rehab, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.

[9 ] Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.

[10 ] Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA.

[11 ] McKusick-Nathans Institute of Genetic Medicine and Department of Genetic Medicine, Johns Hopkins University, Baltimore, MD.

[12 ] Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, United Kingdom.

[13 ] Hospiat Universitari Quiron Dexeus, ICATME Foundation, Barcelona, Spain.

[14 ] Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH.

[15 ] Medical College of Wisconsin, Milwaukee, WI.

[16 ] Hospital Sant Joan de Déu, Barcelona, Spain.

[17 ] Osaka University Hospital, Osaka, Japan.

[18 ] Nemours Children's Hospital, Delaware, Wilmington, DE.

[19 ] Clinical Genetics, Université Paris Cité, INSERM UMR 1163, Institut Imagine, Hôpital Necker Enfants Maladies, Paris, France.

[20 ] School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey.

[21 ] Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.

[22 ] University of Missouri Columbia, Columbia, MO.

[23 ] Otto-von-Guericke-Universität Magdeburg, Magdeburg, Germany.

[24 ] Saitama Children's Medical Center, Saitama, Japan.

[25 ] Tokushima University Hospital, Tokushima, Japan.

[26 ] Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indiana University, Indianapolis, IN.

[27 ] Seattle Children's Hospital, Seattle, WA.

[28 ] BioMarin Pharmaceutical Inc, Novato, CA.

[29 ] BioMarin (UK) Limited, London, United Kingdom.

Article

Publisher Item ID: S1098-3600(22)00906-6

DOI: 10.1016/j.gim.2022.08.015

PubMed ID: 36107167

SO-VID: 38bb8bc5-53e3-430b-960a-bff5b69842b1

History

Keywords: Annualized growth velocity,Achondroplasia,Pediatrics,Observational,Anthropometrics

Data availability:

Keywords: Annualized growth velocity, Achondroplasia, Pediatrics, Observational, Anthropometrics

Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study.

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