Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retr...
Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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Contents
Enzyme replacement therapy (ERT) with laronidase, (recombinant human α-L-iduronidase; Aldurazyme) is the primary treatment option for patients with attenuated mucopolysaccharidosis type I (MPS I). This study examined the effect of early ERT on clinical manifestations.
This multinational, retrospective case series abstracted data from records of...
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Full title
Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4597395
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4597395
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-015-0344-4
