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Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease.

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第一作者： Maarten,Arends

第一单位： Department of Endocrinology and Metabolism, Academic Medical Center, The Netherlands. Electronic address: m.arends@amc.uva.nl.

作者： Maarten,Arends ^[1] ; Frits A,Wijburg ^[2] ; Christoph,Wanner ^[3] ; Frédéric M,Vaz ^[4] ; André B P,van Kuilenburg ^[5] ; Derralynn A,Hughes ^[6] ; Marieke,Biegstraaten ^[7] ; Atul,Mehta ^[8] ; Carla E M,Hollak ^[9] ; Mirjam,Langeveld ^[10]

作者单位： Department of Endocrinology and Metabolism, Academic Medical Center, The Netherlands. Electronic address: m.arends@amc.uva.nl. ^[1] Department of Pediatrics, Academic Medical Center, The Netherlands. Electronic address: f.a.wijburg@amc.uva.nl. ^[2] Department of Internal Medicine I, Division of Nephrology and Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital Wuerzburg, Germany. Electronic address: wanner_c@ukw.de. ^[3] Laboratory Genetic Metabolic Diseases, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. Electronic address: f.m.vaz@amc.uva.nl. ^[4] Laboratory Genetic Metabolic Diseases, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. Electronic address: a.b.vankuilenburg@amc.uva.nl. ^[5] Department of Haematology, Royal Free London NHS Foundation Trust and University College London, United Kingdom. Electronic address: rmgvdah@ucl.ac.uk. ^[6] Department of Endocrinology and Metabolism, Academic Medical Center, The Netherlands. Electronic address: m.biegstraaten@amc.uva.nl. ^[7] Department of Haematology, Royal Free London NHS Foundation Trust and University College London, United Kingdom. Electronic address: atul.mehta1@nhs.net. ^[8] Department of Endocrinology and Metabolism, Academic Medical Center, The Netherlands. Electronic address: c.e.hollak@amc.uva.nl. ^[9] Department of Endocrinology and Metabolism, Academic Medical Center, The Netherlands. Electronic address: m.langeveld@amc.uva.nl. ^[10]

关键词 Early-treatment Enzyme replacement therapy Fabry disease Globotriaosylsphingosine LysoGb3