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Velmanase alfa

Cat. No. M25456
Velmanase alfa Structure
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Biological Activity

Velmanase alfa is a recombinant human lysosomal alpha-mannosidase. Velmanase alfa has an amino acid sequence of the monomeric protein identical to the naturally occurring human alpha-mannosidase. Velmanase alfa supplements or restores the function of deficient alpha-mannosidase. Alpha-mannosidase is a lysosomal enzyme involved in glycoprotein catabolism. Deficient alpha-mannosidase results in the accumulation of mannose-rich oligosaccharides, causing impaired cellular function and apoptosis in all tissues.

Chemical Information
CAS Number 1492823-75-2
Storage Powder          -20°C   3 years ;  4°C   2 years
In solvent       -80°C   6 months ;  -20°C   1 month
Conversion of different model animals based on BSA (PMID: 27057123)
Species Mouse Rat Rabbit Guinea pig Hamster Dog
Weight (kg) 0.02 0.15 1.8 0.4 0.08 10
Body Surface Area (m2) 0.007 0.025 0.15 0.05 0.02 0.5
Km factor 3 6 12 8 5 20
Animal A (mg/kg) = Animal B (mg/kg) multiplied by  Animal B Km
Animal A Km

For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.

References

[1] Sundus Abdul Ghani, et al. Int J Surg. Can velmanase alfa be the next widespread potential therapy for alpha mannosidosis?

[2] No authors listed. Am J Health Syst Pharm. Velmanase Alfa-tycv

[3] Lucia Santoro, et al. JIMD Rep. Early biochemical effects of velmanase alfa in a 7-month-old infant with alpha-mannosidosis

[4] Paul Harmatz, et al. Mol Genet Metab. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis

[5] Dag Malm, et al. Alpha-Mannosidosis

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