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Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)

BACKGROUND: Mucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X‐linked disease caused by a deficiency of the lysosomal enzyme iduronate‐2‐sulfatase, which catalyses a step in the catabolism of glycosaminoglycans. The glycosaminoglycans accumulate within tissues affecting multiple o...

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Bibliografske podrobnosti
izdano v:Cochrane Database Syst Rev
Main Authors: da Silva, Edina MK, Strufaldi, Maria Wany Louzada, Andriolo, Regis B, Silva, Laercio A
Format: Artigo
Jezik:Inglês
Izdano: John Wiley & Sons, Ltd 2016
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7173756/
https://ncbi.nlm.nih.gov/pubmed/26845288
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD008185.pub4
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