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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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Detalles Bibliográficos
Autores principales:	Yergey, Alfred L., Blank, Paul S., Cologna, Stephanie M., Backlund, Peter S., Porter, Forbes D., Darling, Allan J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2017
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5393605/ https://www.ncbi.nlm.nih.gov/pubmed/28414792 http://dx.doi.org/10.1371/journal.pone.0175478

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