Drug Central

Drug results: 6

velmanase	Velmanase alfa-tycv provides an exogenous source of alpha-mannosidase. Velmanase alfa-tycv is internalized via binding to the mannose-6-phosphate receptor on the cell surface and transported into lysosomes where it is thought to exert enzyme activity. Alpha-mannosidosis is a lysosomal storage disease that results from reduced activity of the enzyme alpha-mannosidase, caused by gene variants in Mannosidase Alpha Class 2B Member 1. Alpha-mannosidase catalyzes the degradation of accumulated mannose-containing oligosaccharides. The deficiency of alpha-mannosidase causes an intra-lysosomal accumulation of mannose-rich oligosaccharides in various tissues.
alglucerase	modified form of human placental glucocerebrosidase; oligosaccharide units of glucocerebrosidase are sequentially deglycosylated to yield a mannose-terminated preparation which is targeted to the mannose lectin on the macrophage plasma membrane
taliglucerase alfa	Taliglucerase alfa, a long term enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. Taliglucerase alfa uptake into cellular lysosomes is mediated by binding of Taliglucerase alfa mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.
cipaglucosidase alfa	Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency). Cipaglucosidase alfa is intended to replace the absent or impaired endogenous enzyme. Cipaglucosidase alfa is stabilised by miglustat minimising the loss of enzyme activity in the blood during infusion of this hydrolytic glycogen-specific enzyme enriched with bis-M6P N-glycans for high affinity cation-independent mannose-6-phosphate receptor (CI-MPR) binding. After binding, it is internalised in the lysosome where it undergoes proteolytic cleavage and N-glycan trimming which are both required to yield the most mature and active form of the GAA enzyme. Cipaglucosidase alfa then exerts enzymatic activity in cleaving glycogen and reducing intramuscular glycogen, and ameliorating tissue damage.
technetium Tc 99m tilmanocept	Tilmanocept is a receptor-targeted radiopharmaceutical that is designed to rapidly transit lymphatic vessels; it biotargets, accumulates, and is retained in primary, key predictive, draining lymph nodes (sentinel lymphnodes). The drug substance, tilmanocept, specifically binds to mannose binding receptor proteins (CD206) that reside on the surface of macrophages and dendritic cells. Macrophages are present in high concentrations in lymph nodes.
vestronidase alfa	Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.

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