Tìm theo
Alglucosidase alfa
Các tên gọi khác (7 ) :
  • Acid maltase
  • Acid-alpha glucosidase
  • Aglucosidase alfa
  • Aglucosidase alpha
  • alpha-1,4-glucosidase
  • Alpha-glucosidase
  • Lysosomal Alpha-Glucosidase
enzyme replacement agents
Thuốc Gốc
Biotech
CAS: 420784-05-0
ATC: A16AB07
ĐG : Genzyme Inc. , http://www.genzyme.ca
CTHH: C4435H6739N1175O1279S32
PTK: 105270.8020
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.
Nhận Dạng Quốc Tế & Đặc Tính Hóa Học
Công thức hóa học
C4435H6739N1175O1279S32
Phân tử khối
105270.8020
Dược Lực Học : 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. Alglucosidase alfa 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.
Cơ Chế Tác Dụng : 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. 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.
Dược Động Học :

▧ Volume of Distribution :
* 96 ± 16 mL/kg [20 mg/kg dose] * 119 ± 28 mL/kg [40 mg/kg dose]
▧ Route of Elimination :
Via kidney and liver
▧ Half Life :
2.3 ± 0.4 hours.
▧ Clearance :
* 25+/- 4 mL/hr/kg [4-hour IV infusion of 20 mg/kg]
Độc Tính : There have been no reports of overdose with alglucosidase alfa.
Chỉ Định : For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
Liều Lượng & Cách Dùng : Powder, for solution - Intravenous
Powder, for solution - Intravenous
Dữ Kiện Thương Mại
Giá thị trường
  • Biệt dược thương mại : Myozyme 50 mg vial
    Giá bán buôn : USD >720.0
    Đơn vị tính : vial
Nhà Sản Xuất
  • Công ty :
    Sản phẩm biệt dược : Lumizyme
  • Công ty : Genzyme
    Sản phẩm biệt dược : Myozyme
Đóng gói
Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB01272
KEGG Drug
http://www.genome.jp/dbget-bin/www_bget?drug:D03207
National Drug Code Directory
http://www.hipaaspace.com/Medical_Billing/Coding/National.Drug.Codes/PDF/58468-0150-1
PharmGKB
http://www.pharmgkb.org/drug/PA164754753
Wikipedia
http://en.wikipedia.org/wiki/Alglucosidase_alfa
RxList
http://www.rxlist.com/cgi/generic/myozyme.htm
Drugs.com
http://www.drugs.com/cdi/alglucosidase-alfa.html
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