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2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1
The rare, chronic, autosomal-recessive lysosomal storage disease Niemann-Pick disease type C1 (NPC1) is characterized by progressively debilitating and ultimately fatal neurological manifestations. There is an urgent need for disease-modifying therapies that address NPC1 neurological pathophysiology...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Bentham Science Publishers
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5824462/ https://www.ncbi.nlm.nih.gov/pubmed/29065825 http://dx.doi.org/10.2174/1381612823666171019164220 |