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...
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| Format: | Book |
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"Paediatrician" Publishers LLC,
2020-12-01T00:00:00Z.
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A1234.567 |
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