Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)

BACKGROUND: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare, X-linked disorder caused by deficient activity of the enzyme iduronate-2-sulfatase (I2S). Treatment is available in the form of enzyme replacement therapy (ERT) with recombinant I2S. Clinical outcomes following ≥3 years o...

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Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Muenzer, Joseph, Giugliani, Roberto, Scarpa, Maurizio, Tylki-Szymańska, Anna, Jego, Virginie, Beck, Michael
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2017
Témata:
Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5627440/
https://ncbi.nlm.nih.gov/pubmed/28974237
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0712-3
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