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Using a Novel Measuring Device to Objectively Evaluate the Clinical Benefit on Ptosis in Infantile-Onset Pompe Disease (IOPD): Results from 145 Weeks' Avalglucosidase Alfa Dosing in Mini-COMET Participants Previously Treated with Alglucosidase Alfa

Priya S. Kishnani,Marguerite C. Weinert,James Davison,Anais Brassier,Alexander Broomfield,Yin-Hsiu Chien,Sihoun Hahn,David Kronn,Satoko Kumada,Francois Labarthe,Hirotaka Ohki,Kristina An Haack,Olivier Huynh-Ba,Susan Sparks,Swathi Tammireddy,Tianyue Zhou,S. Grace Prakalapakorn

MOLECULAR GENETICS AND METABOLISM（2024）

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