Experience of Idursulfase Beta Administration in the Child with Mucopolysaccharidosis Type II: Clinical Case
Background. Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare hereditary lysosomal storage disease associated with iduronate-2-sulfatase deficiency. Patients with MPS II require life-long enzyme replacement therapy (ERT) to replace the deficiency of endogenous enzyme. There are two m...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
"Paediatrician" Publishers LLC
2020-12-01
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| Series: | Voprosy Sovremennoj Pediatrii |
| Subjects: | |
| Online Access: | https://vsp.spr-journal.ru/jour/article/view/2497 |