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4. Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease. Nicoli ER; Al Eisa N; Cluzeau CV; Wassif CA; Gray J; Burkert KR; Smith DA; Morris L; Cologna SM; Peer CJ; Sissung TM; Uscatu CD; Figg WD; Pavan WJ; Vite CH; Porter FD; Platt FM PLoS One; 2016; 11(3):e0152007. PubMed ID: 27019000 [TBL] [Abstract][Full Text] [Related]
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12. Altered transition metal homeostasis in Niemann-Pick disease, type C1. Hung YH; Faux NG; Killilea DW; Yanjanin N; Firnkes S; Volitakis I; Ganio G; Walterfang M; Hastings C; Porter FD; Ory DS; Bush AI Metallomics; 2014 Mar; 6(3):542-53. PubMed ID: 24343124 [TBL] [Abstract][Full Text] [Related]
13. Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. Hastings C; Liu B; Hurst B; Cox GF; Hrynkow S Mol Genet Metab; 2022 Dec; 137(4):309-319. PubMed ID: 36279795 [TBL] [Abstract][Full Text] [Related]
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15. Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease. Vite CH; Bagel JH; Swain GP; Prociuk M; Sikora TU; Stein VM; O'Donnell P; Ruane T; Ward S; Crooks A; Li S; Mauldin E; Stellar S; De Meulder M; Kao ML; Ory DS; Davidson C; Vanier MT; Walkley SU Sci Transl Med; 2015 Feb; 7(276):276ra26. PubMed ID: 25717099 [TBL] [Abstract][Full Text] [Related]
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