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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...

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Detalles Bibliográficos
Autores principales: Serratrice, Christine, Swiader, Laure, Serratrice, Jacques
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
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