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Vestronidase Alfa: A Review in Mucopolysaccharidosis VII

Mucopolysaccharidosis VII is an extremely rare, autosomal recessive lysosomal storage disorder characterized by a deficiency of β-glucuronidase activity, resulting in partial degradation and accumulation of GAGs in numerous tissues throughout the body, with consequent cellular damage and organ dysfu...

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Shranjeno v:
Bibliografske podrobnosti
izdano v:BioDrugs
Main Authors: McCafferty, Emma H., Scott, Lesley J.
Format: Artigo
Jezik:Inglês
Izdano: Springer International Publishing 2019
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6469592/
https://ncbi.nlm.nih.gov/pubmed/30848434
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40259-019-00344-7
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