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 CSF 24(S)-HC concentrations in three participants given either 600 or 900 mg of HPbetaCD were increased about two fold (p=0 0032) after drug administration. 2-Hydroxypropyl-beta-cyclodextrin colony stimulating factor 2 Homo sapiens