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- A 32-Year-Old Young Woman with LOPD Successfully Managed with Avalglucosidase Alfa

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Background: Pompe disease is a rare, progressive neuromuscular disorder caused by a deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA), resulting in glycogen accumulation primarily in skeletal and cardiac muscle (1,2). Enzyme replacement therapy (ERT) has been the cornerstone of treatment for Pompe disease, with alglucosidase alfa as t

A 32-Year-Old Young Woman with LOPD Successfully Managed with Avalglucosidase Alfa
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