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...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
Serbian Genetics Society
2018-01-01
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Series: | Genetika |
Subjects: | |
Online Access: | http://www.doiserbia.nb.rs/img/doi/0534-0012/2018/0534-00121803829A.pdf |