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Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I

Enzyme replacement therapy with laronidase (recombinant human alpha-l-iduronidase) is successfully used to treat patients with mucopolysaccharidosis type I (MPS I). However, the intravenously-administered enzyme is not expected to treat or prevent neurological deterioration. As MPS I patients suffer...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Dickson, Patricia I., Kaitila, Ilkka, Harmatz, Paul, Mlikotic, Anton, Chen, Agnes H., Victoroff, Alla, Passage, Merry B., Madden, Jacqueline, Le, Steven Q., Naylor, David E.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4572891/
https://ncbi.nlm.nih.gov/pubmed/26260077
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.07.005
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