Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study

Central nervous system manifestations of mucopolysaccharidosis type I (MPS I) such as cognitive impairment, hydrocephalus, and spinal cord compression are inadequately treated by intravenously-administered enzyme replacement therapy with laronidase (recombinant human alpha-L-iduronidase). While hema...

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發表在:Mol Genet Metab
Main Authors: Chen, Agnes H., Harmatz, Paul, Nestrasil, Igor, Eisengart, Julie B., King, Kelly E., Rudser, Kyle, Kaizer, Alexander M., Svatkova, Alena, Wakumoto, Amy, Le, Steven Q., Madden, Jacqueline, Young, Sarah, Zhang, Haoyue, Polgreen, Lynda E., Dickson, Patricia I.
格式: Artigo
語言:Inglês
出版: 2019
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7813548/
https://ncbi.nlm.nih.gov/pubmed/31839529
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2019.11.007
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