Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation Results in Patients with Hurler Syndrome: Clinical Cases
Mucopolysaccharidosis type I (MPS I) is the hereditary disease characterized with alpha-L-iduronidase activity decrease and further accumulation of heparan and dermatan sulfate in lysosomes. MPS I is rare autosomal recessive disorder with incidence of 0.5-4 cases on 100.000 live-birth infants. Meant...
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| Main Authors: | , , , , , |
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| Format: | Book |
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"Paediatrician" Publishers LLC,
2019-09-01T00:00:00Z.
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