Pompe disease is an inherited lysosomal storage disorder caused by a deficiency in acid alpha-glucosidase (GAA) activity, and is characterized by progressive accumulation of lysosomal glycogen in cardiac and skeletal muscles. Enzyme replacement therapy using recombinant human GAA (rhGAA), alglucosidase alfa, is currently the only approved treatment for Pompe disease. However, alglucosidase alfa may have limitations in delivery to skeletal muscles due to sub-optimal levels of mannose-6-phosphate (M6P), a carbohydrate that binds cation-independent M6P receptors at the cell surface to mediate enzyme internalization and lysosomal delivery.

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