Results of enzyme replacement therapy in Bulgarian patients with a severe form of hunter syndrome: A 42-month follow-up

Hunter syndrome (Mucopolysaccharidosis type II, MPS II) is a rare X-linked disease caused by a deficiency of the enzyme iduronate-2-sulphatase (IDS), which results in the lysosomal accumulation of the undegraded glycosaminoglycans (GAGs) dermatan and heparan sulfate in various tissues an...

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Bibliographic Details
Main Authors: Avdjieva-Tzavella Daniela, Kathom Hadil, Sinigerska Ivanka, Taseva Zlatina, Tincheva Radka
Format: Article
Language:English
Published: Serbian Genetics Society 2018-01-01
Series:Genetika
Subjects:
Online Access:http://www.doiserbia.nb.rs/img/doi/0534-0012/2018/0534-00121803829A.pdf