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

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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Bibliographic Details
Main Authors: Mojca Zerjav Tansek, Jana Kodric, Simona Klemencic, Jaap Jan Boelens, Peter M. van Hasselt, Ana Drole Torkar, Maja Doric, Alenka Koren, Simona Avcin, Tadej Battelino, Urh Groselj
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
MPS
Mucopolysaccharidosis type II
Hunter syndrome
Hematopoietic stem cell transplantation
HSCT, enzyme replacement therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426921000732

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