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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Detalles Bibliográficos
Autores principales: McCafferty, Emma H., Scott, Lesley J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2019
Materias:
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