Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1

2-Hydroxypropyl-beta-cyclodextrin (HPβCD) has gained recent attention as a potential therapeutic intervention in the treatment of the rare autosomal-recessive, neurodegenerative lysosomal storage disorder Niemann-Pick Disease Type C1 (NPC1). Notably, HPβCD formulations are not comprised of a single...

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Bibliographic Details
Published in:PLoS One
Main Authors: Yergey, Alfred L., Blank, Paul S., Cologna, Stephanie M., Backlund, Peter S., Porter, Forbes D., Darling, Allan J.
Format: Artigo
Language:Inglês
Published: Public Library of Science 2017
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5393605/
https://ncbi.nlm.nih.gov/pubmed/28414792
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0175478
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