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Spotlight on taliglucerase alfa in the treatment of pediatric patients with type 1 Gaucher disease
Gaucher disease (GD) is a heritable storage disorder caused by functional defects of the lysosomal acid β-glucosidase and the accumulation of glucosylceramide within macrophages, resulting in multiple organ dysfunction. There are three commercially available enzyme replacement therapy (ERT) products...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Dove Medical Press
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5774592/ https://www.ncbi.nlm.nih.gov/pubmed/29388611 http://dx.doi.org/10.2147/PHMT.S93634 |