Cargando…
Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease-specific treatment is available in the form of enzyme replacement therapy wit...
Autores principales: | Muenzer, Joseph, Jones, Simon A., Tylki-Szymańska, Anna, Harmatz, Paul, Mendelsohn, Nancy J., Guffon, Nathalie, Giugliani, Roberto, Burton, Barbara K., Scarpa, Maurizio, Beck, Michael, Jangelind, Yvonne, Hernberg-Stahl, Elizabeth, Larsen, Maria Paabøl, Pulles, Tom, Whiteman, David A. H. |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2017
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5414331/ https://www.ncbi.nlm.nih.gov/pubmed/28464912 http://dx.doi.org/10.1186/s13023-017-0635-z |
Ejemplares similares
-
Clinical outcomes in idursulfase-treated
patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey
(HOS)
por: Muenzer, Joseph, et al.
Publicado: (2017) -
Birth weight in patients with mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS)
por: Bodamer, Olaf, et al.
Publicado: (2017) -
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)
por: Muenzer, Joseph, et al.
Publicado: (2021) -
Intrafamilial variability in the clinical manifestations of mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS)
por: Ficicioglu, Can, et al.
Publicado: (2017) -
The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
por: Muenzer, Joseph, et al.
Publicado: (2011)