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

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Title: Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I.
Authors: Kuiper GA; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Nijmeijer SCM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Roelofs MJM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands., van der Lee JH; Amsterdam UMC, University of Amsterdam, Pediatric Clinical Research Office, Meibergdreef 9, Amsterdam, Netherlands., Hollak CEM; Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Bosch AM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2019 Sep; Vol. 42 (5), pp. 762-775. Date of Electronic Publication: 2019 May 16.
Publication Type: Journal Article; Systematic Review
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I.
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  Data: <searchLink fieldCode="AU" term="%22Kuiper+GA%22">Kuiper GA</searchLink>; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22Nijmeijer+SCM%22">Nijmeijer SCM</searchLink>; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22Roelofs+MJM%22">Roelofs MJM</searchLink>; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22van+der+Lee+JH%22">van der Lee JH</searchLink>; Amsterdam UMC, University of Amsterdam, Pediatric Clinical Research Office, Meibergdreef 9, Amsterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22Hollak+CEM%22">Hollak CEM</searchLink>; Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22Bosch+AM%22">Bosch AM</searchLink>; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands.
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  Data: <searchLink fieldCode="JN" term="%227910918%22">Journal of inherited metabolic disease</searchLink> [J Inherit Metab Dis] 2019 Sep; Vol. 42 (5), pp. 762-775. <i>Date of Electronic Publication: </i>2019 May 16.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Wiley%22">Wiley </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>7910918 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1573-2665 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2201418955%22">01418955 </searchLink><i>NLM ISO Abbreviation: </i>J Inherit Metab Dis <i>Subsets: </i>MEDLINE
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        Value: 10.1002/jimd.12103
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        Text: English
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              Text: 2019 Sep
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