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Namespace Prefixes

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Statements

Subject Item
n2:DB01272
rdf:type
n3:Drug
n3:description
Aglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes. It is encoded by the most predominant of nine observed haplotypes of this gene. Aglucosidase alfa is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Alglucosidase alfa degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen. Structurally, Alglucosidase alfa is a glycoprotein with a calculated mass of 98,008 daltons for the 883 residue mature polypeptide chain, and a total mass of approximately 109,000 daltons, including carbohydrates. It is used for the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
n3:dosage
n4:271B61B8-363D-11E5-9242-09173F13E4C5 n4:271B61B9-363D-11E5-9242-09173F13E4C5 n4:271B61B7-363D-11E5-9242-09173F13E4C5
n3:generalReferences
# Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE: Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 2007 Jan 9;68(2):99-109. Epub 2006 Dec 6. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17151339
n3:group
approved
n3:halfLife
2.3 ± 0.4 hours.
n3:indication
For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
owl:sameAs
n15:DB01272 n24:DB01272
dcterms:title
Alglucosidase alfa
adms:identifier
n9:D03207 n12:DB01272 n13:58468-0150-1 n20:Alglucosidase_alfa n21:PA164754753
n3:mechanismOfAction
Alglucosidase alfa is designed to act as an exogenous source of GAA, acting to correct GAA deficiency that is the hallmark of Pompe disease. Alglucosidase alfa binds to mannose-6-phosphate receptors on the cell surface via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen. Specifically, it hydrolyses alpha-1,4-glucose bonds.
n3:packager
n16:271B61B5-363D-11E5-9242-09173F13E4C5
n3:patent
n5:2416492
n3:routeOfElimination
Via kidney and liver
n3:synonym
Aglucosidase alpha alpha-1,4-glucosidase Alpha-glucosidase Aglucosidase alfa Acid-alpha glucosidase Acid maltase Lysosomal Alpha-Glucosidase
n3:toxicity
There have been no reports of overdose with alglucosidase alfa.
n3:volumeOfDistribution
* 96 ± 16 mL/kg [20 mg/kg dose] * 119 ± 28 mL/kg [40 mg/kg dose]
n6:hasAHFSCode
n7:44-00-00
foaf:page
n11:myozyme.htm n19:alglucosidase-alfa.html
n3:Molecular-Formula
n18:271B61BB-363D-11E5-9242-09173F13E4C5
n3:Molecular-Weight
n18:271B61BA-363D-11E5-9242-09173F13E4C5
n6:hasATCCode
n22:A16AB07
n3:affectedOrganism
Humans and other mammals
n3:casRegistryNumber
420784-05-0
n3:category
n3:clearance
* 25+/- 4 mL/hr/kg [4-hour IV infusion of 20 mg/kg]
n3:containedIn
n17:271B61B6-363D-11E5-9242-09173F13E4C5