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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2019
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6469592/ https://www.ncbi.nlm.nih.gov/pubmed/30848434 http://dx.doi.org/10.1007/s40259-019-00344-7 |