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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...
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| Publicado no: | Pediatric Health Med Ther |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Dove Medical Press
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5774592/ https://ncbi.nlm.nih.gov/pubmed/29388611 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/PHMT.S93634 |
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