avalglucosidase alfa (Pending FDA Approval)

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Dosing & Uses

Pompe Disease

Pending FDA approval for glycogen storage type II disease (Pompe disease)

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Pharmacology

Mechanism of Action

Enzyme replacement therapy

Alpha-glucosidase is essential for normal muscle development and function; it binds to mannose-6-phosphate receptors and then is transported into lysosomes and undergoes proteolytic cleavage that results in increased enzymatic activity and ability to cleave glycogen

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.