elosulfase alfa

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elosulfase alfa

GtoPdb Ligand ID: 7392

Synonyms: BMN 110 | rhGALNS | Vimizim®

elosulfase alfa is an approved drug (EMA & FDA (2014))

Comment: Elosulfase alfa is a recombinant enzyme replacement therapy for galactosamine (N-acetyl)-6-sulfatase (GALNS, P34059) deficiency. The peptide is translated from the human cDNA expressed in CHO cells [1].

Summary
Biological activity
Clinical data
References
Structure

References
1. Dvorak-Ewell M, Wendt D, Hague C, Christianson T, Koppaka V, Crippen D, Kakkis E, Vellard M. (2010) Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice. PLoS ONE, 5 (8): e12194. [PMID:20808938]

References

1. Dvorak-Ewell M, Wendt D, Hague C, Christianson T, Koppaka V, Crippen D, Kakkis E, Vellard M. (2010)
Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice.
PLoS ONE, 5 (8): e12194. [PMID:20808938]