Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

BACKGROUND: Mucopolysaccharidosis II (MPS II; Hunter syndrome) is a rare, life-limiting lysosomal storage disease caused by deficient iduronate-2-sulfatase activity. Enzyme replacement therapy (ERT) with intravenous (IV) idursulfase can stabilize or improve many somatic manifestations, but there rem...

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Detalles Bibliográficos
Autores principales: Muenzer, Joseph, Botha, Jaco, Harmatz, Paul, Giugliani, Roberto, Kampmann, Christoph, Burton, Barbara K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8557006/
https://www.ncbi.nlm.nih.gov/pubmed/34717704
http://dx.doi.org/10.1186/s13023-021-02052-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8557006/
https://www.ncbi.nlm.nih.gov/pubmed/34717704
http://dx.doi.org/10.1186/s13023-021-02052-4

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