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Mini-COMET Study: Safety and Efficacy Data after Avalglucosidase Alfa Dosing for ≥ 145 Weeks in Participants with Infantile-Onset Pompe Disease (IOPD) Who Had Demonstrated Clinical Decline or Sub-Optimal Response While Receiving Alglucosidase Alfa

David Kronn,Priya Kishnani,James Davison,Anais Brassier,Alexander Broomfield,Sihoun Hahn,Satoko Kumada,Francois Labarthe,Hirotaka Ohki,Kristina An Haack,Susan Sparks,Swathi Tammireddy,Atef Zaher,Tianyue Zhou,Yin-Hsiu Chien

Molecular Genetics and Metabolism（2024）

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Pediatric Feeding Disorders
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