Hematopoietic Stem Cell Transplantation in Mucopolysaccharidosis Type II
Mucopolysaccharidosis II (MPS II—Hunter syndrome) is an X-linked lysosomal storage disorder caused by a deficiency in iduronate-2 sulfatase. Enzyme replacement therapy does not cross the blood–brain barrier (BBB), limiting the results in neurological forms of the disease. Another treatment option fo...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
SciELO
2018-05-01
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| Series: | Journal of Inborn Errors of Metabolism and Screening |
| Online Access: | https://doi.org/10.1177/2326409818779097 |