The natural history of MPS I: global perspectives from the MPS I Registry

PURPOSE: In this study, we aimed to describe the natural history of mucopolysaccharidosis I. METHODS: Data from 1,046 patients who enrolled in the MPS I Registry as of August 2013 were available for descriptive analysis. Only data from untreated patients and data prior to treatment for patients who...

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Detalhes bibliográficos
Main Authors: Beck, Michael, Arn, Pamela, Giugliani, Roberto, Muenzer, Joseph, Okuyama, Torayuki, Taylor, John, Fallet, Shari
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4189384/
https://ncbi.nlm.nih.gov/pubmed/24675674
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2014.25
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