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Pharmacokinetics and distribution of 2‐hydroxypropyl‐β‐cyclodextrin following a single intrathecal dose to cats

2‐Hydroxypropyl‐β‐cyclodextrin (HP‐β‐CD) is an experimental therapy for Niemann‐Pick disease type C (NPC) that reduced neuronal cholesterol and ganglioside storage, reduced Purkinje cell death, and increased lifespan in npc1−/− mice and NPC1 cats. In this study, tissue distribution was investigated...

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Detalles Bibliográficos
Autores principales: Kao, Mark L., Stellar, Susan, Solon, Eric, Lordi, Alfred, Kasica, Nicole, Swain, Gary, Bagel, Jessica H., Gurda, Brittney L., Vite, Charles H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7317750/
https://www.ncbi.nlm.nih.gov/pubmed/31707730
http://dx.doi.org/10.1002/jimd.12189