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Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease

OBJECTIVE: Niemann–Pick type C (NPC) disease is a fatal, neurodegenerative, lysosomal storage disorder characterized by intracellular accumulation of unesterified cholesterol (UC) and other lipids. While its mechanism of action remains unresolved, administration of 2‐hydroxypropyl‐β‐cyclodextrin (HP...

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Detalles Bibliográficos
Autores principales:	Davidson, Cristin D., Fishman, Yonatan I., Puskás, István, Szemán, Julianna, Sohajda, Tamás, McCauliff, Leslie A., Sikora, Jakub, Storch, Judith, Vanier, Marie T., Szente, Lajos, Walkley, Steven U., Dobrenis, Kostantin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2016
Materias:	Research Papers
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4863749/ https://www.ncbi.nlm.nih.gov/pubmed/27231706 http://dx.doi.org/10.1002/acn3.306

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