Therapy-type related long-term outcomes in mucopolysaccaridosis type II (Hunter syndrome) – Case series

Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare, X-linked recessive multisystem lysosomal storage disease due to iduronate-2-sulfatase enzyme deficiency. We presented three unrelated Slovenian patients with the severe form of MPS II that received three different management approach...

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Detalles Bibliográficos
Autores principales: Zerjav Tansek, Mojca, Kodric, Jana, Klemencic, Simona, Boelens, Jaap Jan, van Hasselt, Peter M., Drole Torkar, Ana, Doric, Maja, Koren, Alenka, Avcin, Simona, Battelino, Tadej, Groselj, Urh
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8251508/
https://www.ncbi.nlm.nih.gov/pubmed/34258227
http://dx.doi.org/10.1016/j.ymgmr.2021.100779

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8251508/
https://www.ncbi.nlm.nih.gov/pubmed/34258227
http://dx.doi.org/10.1016/j.ymgmr.2021.100779

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