Idursulfase Beta (Hunterase) Therapeutic Experience in a Patient with Mucopolysaccharidosis Type II
Background. Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-linked recessive disease caused by lysosomal enzyme iduronate-2-sulfatase deficiency resulting in progressive glycosaminoglycans (GAG) accumulation in tissues (dermatan sulfate and heparan sulfate). GAG accumulation in cells...
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Main Authors: | Nato D. Vashakmadze (Author), Nataliya V. Zhurkova (Author), Ekaterina Yu. Zakharova (Author), Ludmila K. Mikhaylova (Author), Marina A. Babaykina (Author) |
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Format: | Book |
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"Paediatrician" Publishers LLC,
2023-08-01T00:00:00Z.
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