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

Caroline A. Hastings, Sharon Hrynkow|SSRN Electronic Journal|2022

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