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Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.
Savarirayan, Ravi; Tofts, Louise; Irving, Melita; Wilcox, William; Bacino, Carlos A; Hoover-Fong, Julie; Ullot Font, Rosendo; Harmatz, Paul; Rutsch, Frank; Bober, Michael B; Polgreen, Lynda E; Ginebreda, Ignacio; Mohnike, Klaus; Charrow, Joel; Hoernschemeyer, Daniel; Ozono, Keiichi; Alanay, Yasemin; Arundel, Paul; Kagami, Shoji; Yasui, Natsuo; White, Klane K; Saal, Howard M; Leiva-Gea, Antonio; Luna-González, Felipe; Mochizuki, Hiroshi; Basel, Donald; Porco, Dania M; Jayaram, Kala; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan.
Afiliación
  • 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.
Lancet ; 396(10252): 684-692, 2020 09 05.
Article en En | MEDLINE | ID: mdl-32891212
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Osteogénesis / Acondroplasia / Péptido Natriurético Tipo-C Tipo de estudio: Clinical_trials Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Lancet Año: 2020 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Osteogénesis / Acondroplasia / Péptido Natriurético Tipo-C Tipo de estudio: Clinical_trials Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Lancet Año: 2020 Tipo del documento: Article