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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
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Article
Acesso em linha: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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