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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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| Udgivet i: | BioDrugs |
|---|---|
| Main Authors: | , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Springer International Publishing
2019
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| Fag: | |
| Online adgang: | 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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