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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...
Autores principales: | Pasqualim, Gabriela, Baldo, Guilherme, de Carvalho, Talita Giacomet, Tavares, Angela Maria Vicente, Giugliani, Roberto, Matte, Ursula |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2015
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Materias: | |
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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