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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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Detalles Bibliográficos
Autores principales:	Pasqualim, Gabriela, Baldo, Guilherme, de Carvalho, Talita Giacomet, Tavares, Angela Maria Vicente, Giugliani, Roberto, Matte, Ursula
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2015
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4315431/ https://www.ncbi.nlm.nih.gov/pubmed/25646802 http://dx.doi.org/10.1371/journal.pone.0117271

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