Zobrazeno 1 - 1
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pro vyhledávání: '"Dafne D. G. Horovitz MD, PhD"'
Publikováno v:
Journal of Inborn Errors of Metabolism and Screening, Vol 6 (2018)
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 neurolog
Externí odkaz:
https://doaj.org/article/046d49182b824391aee55e008044d0c3
