Alglucosidase alfa (Lumizyme Myozyme Genzyme) is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II) a rare lysosomal storage disorder (LSD).Chemically speaking the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease alpha-glucosidase. It is the first drug available to treat this disease.
ATC prefix
A16
ATC suffix
AB07
CAS number
420794-05-0
DrugBank
DB01272
FDA UNII code
DTI67O9503
IUPAC name
Human glucosidase prepro-α-[199-arginine223-histidine]
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