Title : Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.

Pub. Date : 2017 Oct 14

PMID : 28803710






1 Functional Relationships(s)
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1 METHODS: In this open-label, dose-escalation phase 1-2a study, we gave monthly intrathecal HPbetaCD to participants with NPC1 with neurological manifestation at the National Institutes of Health (NIH), Bethesda, MD, USA. 2-Hydroxypropyl-beta-cyclodextrin NPC intracellular cholesterol transporter 1 Homo sapiens