Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
CORRECTION article
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Provisionally accepted- 1 University of Florida, Gainesville, Florida, United States
- 2 Metabolic Unit, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Campania, Italy
- 3 Friedrich-Baur-Institute, Department of Neurology, LMU University Clinic, LMU Munich, Munich, Bavaria, Germany
- 4 Erasmus MC University Medical Center, Rotterdam, Netherlands
- 5 M6P-Therapeutics, St Louis, United States
- 6 Amicus Therapeutics, Inc., Princeton, New Jersey, United States
- 7 Metrum Research Group, Tariffville, CT, United States
- 8 Incyte Corporation, Wilmington, Delaware, United States
- 9 Amicus Therapeutics (United States), Cranbury, United States
- 10 Department of Neurology, University of California, Irvine, CA, United States
Corrigendum on: Byrne BJ, Parenti G, Schoser B, van der Ploeg AT, Do H, Fox B, Goldman M, Johnson FK, Kang J, Mehta N, Mondick J, Sheikh MO, Sitaraman Das S, Tuske S, Brudvig J, Weimer JM, Mozaffar T. Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease. Front Neurol. 2024Oct 18:15:1451512. doi: 10.3389/fneur.2024.1451512. In the published article, there was an error in Figure 13 as published. The error relates to the positive/negative value of some of the numbers on the axes for the lower MMT score and the GSGC total score in Figure 13 A. For both lower MMT score and GSGC total score, the axis scale incorrectly read -3, -2, 1, 0, -1, -2, -3 from left to right, whereas the axis scale for lower MMT score should have been -3, -2, -1, 0, 1, 2, 3 and the axis scale for GSGC total score should have been 3, 2, 1, 0, -1, -2, -3. The corrected Figure 13 and its caption **Change from baseline at week 52 of PROPELeffect 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. appear below.The authors apologize for this error and state that this does not change the scientific conclusions of the article in any way. The original article has been updated.Reminder: Figures, tables, and images will be published under a Creative Commons CC-BY licence and permission must be obtained for use of copyrighted material from other sources (including re-published/adapted/modified/partial figures and images from the internet). It is the responsibility of the authors to acquire the licenses, to follow any citation instructions requested by third-party rights holders, and cover any supplementary charges.End of template, if you would like to request a correction for a reason not seen here, please contact the journal's Editorial Office
Keywords: Pompe disease, Glycogen Storage Disease Type II, lysosomal storage disorders, Enzyme Replacement Therapy, N-butyldeoxynojirimycin
Received: 05 Dec 2024; Accepted: 12 Dec 2024.
Copyright: © 2024 Byrne, Parenti, Schoser, van der Ploeg, Do, Fox, Goldman, Johnson, Kang, Mehta, Mondick, Sheikh, Sitaraman Das, Tuske, Brudvig, Weimer and Mozaffar. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Barry Byrne, University of Florida, Gainesville, 32609, Florida, United States
Jon Brudvig, Amicus Therapeutics (United States), Cranbury, United States
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