Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease

Abstract

[This corrects the article DOI: 10.3389/fneur.2024.1451512.].

Keywords: Pompe disease; enzyme replacement therapy; glycogen storage disease type II; lysosomal storage disorders; n-butyldeoxynojirimycin.

Figure 13

Change from baseline at week 52 of PROPEL—effect of cipaglucosidase alfa plus miglustat compared with alglucosidase alfa plus placebo in key efficacy outcomes. (A) Forest plot illustrating mean estimated treatment differences between cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo and corresponding 95% CIs are shown for the combined PROPEL study population for each outcome, with units as indicated on the x-axes. For all outcomes, right-sided directionality of treatment differences indicates favorable outcomes for cipaglucosidase alfa plus miglustat compared with alglucosidase alfa plus placebo. (B) The table shows baseline mean values and Week 52 CFBL values for cipaglucosidase alfa plus miglustat and alglucosidase alfa plus placebo. Shaded CFBL indicates nominally significant improvement (green) or nominally significant worsening (red) from baseline (i.e., the 95% CI does not include zero) within each treatment group. The p-values (two-tailed LS mean difference) shown in the far-right column are for the between-group treatment differences illustrated in the forest plot.