Fig. 1

Liver function tests remained in the near-normal range in five Wolman disease (WD) patients treated with sebelipase alfa. A Alanine transaminase (ALT), B aspartate transaminase (AST), C gamma-glutamyltranspeptidase (γGT), D total bilirubin, E albumin and F international normalized ratio (INR) measured in Patients 1–5 (P#1–P#5), before (black) and under (orange) enzyme replacement therapy (ERT). ALT, AST and γGT are expressed as number of times of the upper limit of the normal (xULN). The grey zones and the dotted lines represent the normal values and their limits, respectively. Mann–Whitney test pre versus post, median ± interquartile range, n.s. not significant