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. 2014 Feb 25;137(5):1481–1495. doi: 10.1093/brain/awu020

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© The Author (2014). Published by Oxford University Press on behalf of the Guarantors of Brain.

This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.

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Ambroxol treatment significantly increases glucosylceramidase protein levels. Representative western blots showing increased glucosylceramidase protein levels and decreased cathepsin D levels with ambroxol treatment. ‘+’ represents lanes treated with ambroxol. Ambroxol treatment resulted in a significant elevation of glucosylceramidase protein levels in control [median increase 30% of untreated cells (IQR 15–40%), Mann-Whitney U-test P = 0.0085], Gaucher disease (GD) [median increase 100% of untreated (IQR 87–200% increase), P = 0.004], Parkinson’s disease with GBA mutations (PD-GBA) [median increase 50% of untreated (IQR 40–130%), P = 0.04] and non-manifesting carrier (NMC) fibroblasts [median increase 35% of untreated cells (IQR 25–40% increase), P = 0.008]. Results are derived from all cell lines in each group and represent median value and IQR.