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Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry

Our objective was to assess how the diagnosis and treatment of mucopolysaccharidosis I (MPS I) have changed over time. We used data from 891 patients in the MPS I Registry, an international observational database, to analyze ages at symptom onset, diagnosis, treatment initiation, and treatment alloc...

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Autors principals: D’Aco, Kristin, Underhill, Lisa, Rangachari, Lakshmi, Arn, Pamela, Cox, Gerald F., Giugliani, Roberto, Okuyama, Torayuki, Wijburg, Frits, Kaplan, Paige
Format: Artigo
Idioma:Inglês
Publicat: Springer-Verlag 2012
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3357468/
https://ncbi.nlm.nih.gov/pubmed/22234477
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00431-011-1644-x
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