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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Опубликовано в: :Ann Clin Transl Neurol
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: John Wiley and Sons Inc. 2016
Предметы:
Research Papers
Online-ссылка: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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