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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Detalles Bibliográficos
Autores principales: Gupta, Punita, Pastores, Gregory M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2017
Materias:
Review
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

Internet

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

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