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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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| Udgivet i: | Ann Clin Transl Neurol |
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| Main Authors: | , , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
John Wiley and Sons Inc.
2016
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4863749/ https://ncbi.nlm.nih.gov/pubmed/27231706 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.306 |
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