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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...
Autores principales: | Kao, Mark L., Stellar, Susan, Solon, Eric, Lordi, Alfred, Kasica, Nicole, Swain, Gary, Bagel, Jessica H., Gurda, Brittney L., Vite, Charles H. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2019
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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 |
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