Plasma SPC and GlcSph in NP-C patients and controls.

<p>One sample per patient is shown, where multiple samples from one patient were available the first was used. The dotted horizontal line represents the 95% percentile of the entire control group for SPC (10.3 nM) and GlcSph (2.4 nM). The bar is the median. A and B, SPC and GlcSph in the entire cohort; C and D, SPC and GlcSph separated by age (years); E and F, SPC and GlcSph separated by miglustat status (0–50 years). The miglustat treated patients had been on treatment for 2.8±1.4 years (average ± standard deviation), 0.7–6 years (Min-Max).