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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Detalles Bibliográficos
Autores principales: Muenzer, Joseph, Giugliani, Roberto, Scarpa, Maurizio, Tylki-Szymańska, Anna, Jego, Virginie, Beck, Michael
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5627440/
https://www.ncbi.nlm.nih.gov/pubmed/28974237
http://dx.doi.org/10.1186/s13023-017-0712-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5627440/
https://www.ncbi.nlm.nih.gov/pubmed/28974237
http://dx.doi.org/10.1186/s13023-017-0712-3

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