Mini-COMET Study: Safety, Biomarker, and Efficacy Data After Avalglucosidase Alfa Dosing for >= 97 Weeks in Participants With Infantile-Onset Pompe Disease (IOPD) Previously Treated With Alglucosidase Alfa Who had Demonstrated Clinical Decline
Author Type(s)
Faculty
Document Type
Abstract
Publication Date
2-2022
DOI
10.1016/j.ymgme.2021.11.171
Journal Title
Molecular Genetics and Metabolism
Department
Pediatrics
Recommended Citation
Kronn, D., Davidson, J., Brassier, A., Broomfield, A., Hahn, S. H., Kumada, S., Labarthe, F., Ohki, H., Prakalapakorn, S. G., Haack, K. A., Meng, X. Z., Sparks, S., Tammireddy, S., Wilson, C., Zaher, A., Zhou, T. Y., Chien, Y. H., & Kishnani, P. S. (2022). Mini-COMET Study: Safety, Biomarker, and Efficacy Data After Avalglucosidase Alfa Dosing for >= 97 Weeks in Participants With Infantile-Onset Pompe Disease (IOPD) Previously Treated With Alglucosidase Alfa Who had Demonstrated Clinical Decline. Molecular Genetics and Metabolism, 135 (2), S68-S68. https://doi.org/10.1016/j.ymgme.2021.11.171
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