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Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease

In Gaucher disease (GD), genetic deficiency of acid β-glucosidase leads to accumulation of its substrate glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph). Lipid-laden cells, most prominently seen as macrophages engorged with GlcCer and GlcSph-laden lysosomes, trigger chronic metabolic infl...

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Detalles Bibliográficos
Autores principales:	Kleytman, Nathaniel, Ruan, Jiapeng, Ruan, Audrey, Zhang, Bailin, Murugesan, Vagishwari, Lin, Haiqun, Guo, Lilu, Klinger, Katherine, Mistry, Pramod K.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2021
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8408524/ https://www.ncbi.nlm.nih.gov/pubmed/34485083 http://dx.doi.org/10.1016/j.ymgmr.2021.100798

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