Fig. 5

Changes in height Z-scores over time with olipudase alfa treatment. A Individual height Z-scores over time are shown. Per enrollment criteria, all patients were required to have a height Z-score of − 1.0 or less at baseline. The solid line at Z-score of − 2 indicates cutoff for short stature (height 2 or more standard deviations below the mean for age [20], equating to a Z-score of − 2 or less). A Z-score of 0 represents mean height. B Mean height Z-scores ± standard deviations over time stratified by age category. P values from the ANCOVA test used to examine mean difference from baseline to month 24 are: P = 0.2028 for the adolescent group (n = 3), P = 0.0005 for the child (n = 7), and P = 0.0016 for the infant/early child group (n = 6). Level of significance at each timepoint is indicated in the figure with asterisks (*p < 0.05; **p < 0.01; ***p < 0.001). Summary data at 24 months are provided in Additional file 1: Table S2