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

Internet

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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