Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.-论文-万方医学网

作者： Eveline J,Langereis ^[1] ; Naomi,van Vlies ^[2] ; Heather J,Church ^[3] ; Ronald B,Geskus ^[4] ; Carla E M,Hollak ^[5] ; Simon A,Jones ^[3] ; Wim,Kulik ^[6] ; Henk,van Lenthe ^[6] ; Jean,Mercer ^[3] ; Lena,Schreider ^[7] ; Karen L,Tylee ^[3] ; Tom,Wagemans ^[2] ; Frits A,Wijburg ^[8] ; Brian W,Bigger ^[7]

作者单位： Department of Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. ^[1] Department of Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands; Laboratory for Genetic Metabolic Diseases, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. ^[2] Genetic Medicine, Manchester Academic Health Science Centre, Central Manchester University Hospitals, NHS Foundation Trust, St Mary's Hospital, Manchester, UK. ^[3] Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. ^[4] Department of Internal Medicine, Division of Endocrinology and Metabolism and Amsterdam Lysosome Center 'Sphinx', Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. ^[5] Laboratory for Genetic Metabolic Diseases, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. ^[6] Stem Cell & Neurotherapies, Centre for Genomic Medicine, University of Manchester, Manchester, UK. ^[7] Department of Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands. Electronic address: f.a.wijburg@amc.uva.nl. ^[8]

主题词青少年(Adolescent);成年人(Adult);儿童(Child);儿童, 学龄前(Child, Preschool);硫酸皮肤素(Dermatan Sulfate);二糖类(Disaccharides);酶替代疗法(Enzyme Replacement Therapy);女(雌)性(Female);随访研究(Follow-Up Studies);肝素辅助因子Ⅱ(Heparin Cofactor II);硫酸乙酰肝素(Heparitin Sulfate);人类(Humans);艾杜糖醛酸酶(Iduronidase);免疫球蛋白G(Immunoglobulin G);婴儿(Infant);婴儿, 新生(Infant, Newborn);男(雄)性(Male);黏多糖累积病Ⅰ型(Mucopolysaccharidosis I);重组蛋白质类(Recombinant Proteins);凝血酶(Thrombin);青年人(Young Adult)

DOI 10.1016/j.ymgme.2014.10.012

PMID 25467058

发布时间 2015-11-19

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Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.

Molecular genetics and metabolism

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Molecular genetics and metabolism

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Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.

Molecular genetics and metabolism

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Molecular genetics and metabolism

相关期刊

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