A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II.
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PMID: 25834948
Free article.
Clinical Trial.
Intravenous (i.v.) enzyme replacement therapy does not affect cognitive impairment because recombinant iduronate-2-sulfatase (idursulfase) does not penetrate the blood-brain barrier at therapeutic concentrations. We examined the safety of idursulfase formulat …
Intravenous (i.v.) enzyme replacement therapy does not affect cognitive impairment because recombinant iduronate-2-sulfatase (idursulfase …