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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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Detalles Bibliográficos
Autor principal:	Calias, Pericles
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Bentham Science Publishers 2017
Materias:	Article
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

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