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Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series
INTRODUCTION: Gaucher disease is caused by a deficiency of the enzyme β-glucocerebrosidase. Treatment with enzyme replacement therapy has been available for the past two decades but, although effective, enzyme replacement therapy can be delivered only by intravenous infusion every other week. The or...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2015
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4488047/ https://www.ncbi.nlm.nih.gov/pubmed/26100396 http://dx.doi.org/10.1186/s13256-015-0617-5 |