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Evaluation of the ambroxol effect on the restoration of GCase activity. GCase activity assessed by liquid chromatography coupled with tandem mass spectrometry (LC-MS/MS) in iPSC-derived DA neurons from GBA-PD and GBA-carrier and “healthy” controls before (blue) and after (red) ambroxol (ABX) treatment, p-value < 0.05, n = 12. All experiments were performed on three iPSC lines for each patient.
