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Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model

BACKGROUND: Gaucher disease (GD) is a rare recessively inherited disorder caused by deficiency of a lysosomal enzyme, glucocerebrosidase. Accumulation of glucosylceramide or glucosylsphingosine in macrophages leads to increased production of ferritin and chitotriosidase and to decreases in hemoglobi...

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Detalles Bibliográficos
Autores principales:	Vigan, Marie, Stirnemann, Jérôme, Caillaud, Catherine, Froissart, Roseline, Boutten, Anne, Fantin, Bruno, Belmatoug, Nadia, Mentré, France
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2014
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4094900/ https://www.ncbi.nlm.nih.gov/pubmed/24980507 http://dx.doi.org/10.1186/1750-1172-9-95

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