Miglustat Treatment May Reduce Cerebrospinal Fluid Levels of the Axonal Degeneration Marker Tau in Niemann–Pick Type C

Introduction: Niemann–Pick disease type C (NPC) is a lysosomal storage disorder that leads to progressive neurodegeneration. The glucosylceramide synthase blocker miglustat is being used to treat NPC, but monitoring of disease progression and treatment response is difficult. NPC patients have elevat...

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Autors principals: Mattsson, Niklas, Zetterberg, Henrik, Bianconi, Simona, Yanjanin, Nicole M., Fu, Rao, Månsson, Jan-Eric, Porter, Forbes D., Blennow, Kaj
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2011
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3509856/
https://ncbi.nlm.nih.gov/pubmed/23430872
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2011_47
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