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4. 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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