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- Avalglucosidase Alfa in Late-Onset Pompe Disease: Key Outcomes from the Pivotal Phase 3 COMET Trial

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Medical Article
Introduction: Pompe disease (PD) is a rare, progressive, and often debilitating lysosomal storage disorder caused by mutations in the gene encoding acid alpha-glucosidase (GAA), leading to the accumulation of glycogen in muscle tissues (1). Enzyme replacement therapy (ERT) with recombinant human GAA, such as alglucosidase alfa, has been the primary

Avalglucosidase Alfa in Late-Onset Pompe Disease: Key Outcomes from the Pivotal Phase 3 COMET Trial
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