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Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI.

We report evidence of a dose responsive effect of enzyme replacement therapy in mucopolysaccharidosis type VI cats from birth, at the clinical, biochemical, and histopathological level. Cats treated with weekly, intravenous recombinant human N-acetylgalactosamine-4-sulfatase at 1 and 5 mg/kg, were h...

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Autors principals: Crawley, A C, Niedzielski, K H, Isaac, E L, Davey, R C, Byers, S, Hopwood, J J
Format: Artigo
Idioma:Inglês
Publicat: 1997
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC507847/
https://ncbi.nlm.nih.gov/pubmed/9045867
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