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

Abstract 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...

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Principais autores:	Joseph Muenzer, Roberto Giugliani, Maurizio Scarpa, Anna Tylki-Szymańska, Virginie Jego, Michael Beck
Formato:	Artigo
Idioma:	Inglês
Publicado:	BMC 2017-10-01
Series:	Orphanet Journal of Rare Diseases
Assuntos:	Hunter syndrome Lysosomal storage disease Idursulfase Efficacy Disease registry
Acceso en liña:	http://link.springer.com/article/10.1186/s13023-017-0712-3
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