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Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

BACKGROUND: Mucopolysaccharidosis type I can be classified as three clinical sub‐types; Hurler syndrome, Hurler‐Scheie syndrome and Scheie syndrome, with the scale of severity being such that Hurler syndrome is the most severe and Scheie syndrome the least severe. It is a rare, autosomal recessive d...

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Detalhes bibliográficos
Publicado no:Cochrane Database Syst Rev
Main Authors: Jameson, Elisabeth, Jones, Simon, Remmington, Tracey
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Ltd 2019
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6581069/
https://ncbi.nlm.nih.gov/pubmed/31211405
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD009354.pub5
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