Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I.

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第一作者： Gé-Ann,Kuiper

第一单位： Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center "Sphinx", Meibergdreef 9, Amsterdam, Netherlands.

作者： Gé-Ann,Kuiper ^[1] ; Stephanie C M,Nijmeijer ^[1] ; Manouck J M,Roelofs ^[2] ; Johanneke H,van der Lee ^[3] ; Carla E M,Hollak ^[4] ; Annet M,Bosch ^[2]

作者单位： Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center "Sphinx", Meibergdreef 9, Amsterdam, Netherlands. ^[1] Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands. ^[2] Amsterdam UMC, University of Amsterdam, Pediatric Clinical Research Office, Meibergdreef 9, Amsterdam, Netherlands. ^[3] Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Amsterdam Lysosome Center "Sphinx", Meibergdreef 9, Amsterdam, Netherlands. ^[4]

关键词 effectiveness enzyme replacement therapy laronidase mucopolysaccharidosis type I orphan drug

主题词临床试验(主题)(Clinical Trials as Topic);酶替代疗法(Enzyme Replacement Therapy);人类(Humans);艾杜糖醛酸酶(Iduronidase);黏多糖累积病Ⅰ型(Mucopolysaccharidosis I);生活质量(Quality of Life);治疗结果(Treatment Outcome)

DOI 10.1002/jimd.12103

PMID 31020996

发布时间 2020-09-16

翻译满意度评价：

准确一般字义偏差语意偏离译文作用小提交