Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I (MPS I) is a progressive disorder caused by deficiency of α-L-iduronidase (IDUA), which leads to storage of heparan and dermatan sulphate. It is suggested that early enzyme replacement therapy (ERT) leads to better outcomes, although many patients are diagnosed late and...

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Publicat a:PLoS One
Autors principals: Pasqualim, Gabriela, Baldo, Guilherme, de Carvalho, Talita Giacomet, Tavares, Angela Maria Vicente, Giugliani, Roberto, Matte, Ursula
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2015
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Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4315431/
https://ncbi.nlm.nih.gov/pubmed/25646802
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0117271
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