Acid-alpha Glucosidase

Acid-alpha Glucosidase Uses, Dosage, Side Effects, Food Interaction and all others data.

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. Acid-alpha Glucosidase degrades glycogen by catalyzing the hydrolysis of a-1,4- and a-1,6- glycosidic linkages of lysosomal glycogen. Structurally, Acid-alpha Glucosidase 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.

Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. In the infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac and skeletal muscles, and hepatic tissues, leading to the development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function. In the juvenile- and adult-onset forms, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness. Death in all forms is usually related to respiratory failure. Acid-alpha Glucosidase provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur 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.

Acid-alpha Glucosidase
Uses
Dosage
Side Effect
Precautions
Interactions
Uses during Pregnancy
Uses during Breastfeeding
Accute Overdose
Food Interaction
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Interaction With other Medicine
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Trade Name	Acid-alpha Glucosidase
Generic	Alglucosidase alfa
Alglucosidase alfa Other Names	Acid maltase, Acid-alpha glucosidase, Aglucosidase alfa, Aglucosidase alpha, Alglucosidasa alfa, Alglucosidase alfa, alpha-1,4-glucosidase, Alpha-glucosidase, Lysosomal Alpha-Glucosidase
Type
Formula	C₄₄₃₅H₆₇₃₉N₁₁₇₅O₁₂₇₉S₃₂
Weight	105270.802 Da
Groups	Approved
Therapeutic Class
Manufacturer
Available Country
Last Updated:	September 19, 2023 at 7:00 am

Uses

Acid-alpha Glucosidase is an acid alpha- glucosidase (GAA) derivative used as an enzyme replacement therapy for the treatment of Pompe disease in infants and pediatric patients caused by GAA deficiency.

For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.

Acid-alpha Glucosidase is also used to associated treatment for these conditions: Pompe's Disease

How Acid-alpha Glucosidase works

Acid-alpha Glucosidase is designed to act as an exogenous source of GAA, acting to correct GAA deficiency that is the hallmark of Pompe disease. Acid-alpha Glucosidase 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.