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

Authors

D Kronn, New York Medical College
J Davidson
A Brassier
A Broomfield
S H. Hahn
S Kumada
F Labarthe
H Ohki
S G. Prakalapakorn
K A. Haack
X Z. Meng
S Sparks
S Tammireddy
C Wilson
A Zaher
T Y. Zhou
Y H. Chien
P S. Kishnani

Author Type(s)

Faculty

Document Type

Abstract

Publication Date

2-2022

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