Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII.

Cadaoas, Jaclyn

Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII. [electronic resource] - Molecular genetics and metabolism 05 2020 - 65-76 p. digital

Publication Type: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't

1096-7206

10.1016/j.ymgme.2020.02.009 doi


Administration, Intravenous
Adult
Animals
CHO Cells
Child
Cricetulus
Enzyme Replacement Therapy--methods
Female
Fibroblasts--metabolism
Glucuronidase--administration & dosage
Glycosaminoglycans--metabolism
Humans
Lysosomes--enzymology
Male
Mice
Mice, Transgenic
Mucopolysaccharidosis VII--enzymology
Rats
Rats, Sprague-Dawley
Recombinant Proteins--genetics
Tissue Distribution--drug effects