Tìm theo
Galsulfase
Các tên gọi khác (2) :
  • ARSB
  • Arylsufatase B
enzyme replacement agents
Thuốc Gốc
Biotech
CAS: 552858-79-4
ATC: A16AB08
ĐG : BioMarin Pharmaceuticals Inc. , http://www.bmrn.com
CTHH: C2534H3851N691O719S16
PTK: 56012.6
Galsufase is a variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase of recombinant DNA origin. Galsulfase is a glycoprotein with a molecular weight of approximately 56 kD. The recombinant protein is comprised of 495 amino acids and contains six asparagine-linked glycosylation sites, four of which carry a bis mannose-6-phosphate manose7 oligosaccharide for specific cellular recognition. Post-translational modification of Cys53 produces the catalytic amino acid residue Ca-formylglycine, which is required for enzyme activity and is conserved in all members of the sulfatase enzyme family.
Nhận Dạng Quốc Tế & Đặc Tính Hóa Học
Công thức hóa học
C2534H3851N691O719S16
Phân tử khối
56012.6
Dược Lực Học : Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate dermatan sulfate, throughout the body. This accumulation leads to widespread cellular, tissue, and organ dysfunction. Galsulfase is intended to provide an exogenous enzyme that will be taken up into lysosomes and increase the catabolism of GAG. Galsulfase uptake by cells into lysosomes is most likely mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of galsulfase to specific mannose-6-phosphate receptors.
Cơ Chế Tác Dụng : Galsufase is a variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase of recombinant DNA origin. Galsulfase is a glycoprotein with a molecular weight of approximately 56 kD. The recombinant protein is comprised of 495 amino acids and contains six asparagine-linked glycosylation sites, four of which carry a bis mannose-6-phosphate manose7 oligosaccharide for specific cellular recognition. Post-translational modification of Cys53 produces the catalytic amino acid residue Ca-formylglycine, which is required for enzyme activity and is conserved in all members of the sulfatase enzyme family. Galsulfase supplies recombinant-engineered galsulfase, a normal variant form of the polymorphic human enzyme, N-acetylgalactosamine 4-sulfatase. It is a lysosomal hydrolase that catalyzes the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of GAG chondroitin 4-sulfate and dermatan sulfate. Increased catabolism of GAG in turn reduces systemic dermatan sulfate accumulation, thereby reducing the primary symptoms of MPS VI.
Dược Động Học :

▧ Volume of Distribution :
Week 1: 56-323 mL/kg and 59-2799 mL/kg by week 24
▧ Half Life :
9 (6 to 21) minutes during the first week of treatment, 26 (8 to 40) minutes by the 24th week.
Độc Tính : There is no experience with overdose of galsulfase.
Chỉ Định : For the treatment of adults and children with Mucopolysaccharidosis VI.
Liều Lượng & Cách Dùng : Solution - Intravenous drip
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Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB01279
PharmGKB
http://www.pharmgkb.org/drug/PA164746235
Wikipedia
http://en.wikipedia.org/wiki/Galsulfase
RxList
http://www.rxlist.com/cgi/generic/naglazyme.htm
Drugs.com
http://www.drugs.com/cdi/galsulfase.html
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