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Case Report: Ursodeoxycholic acid treatment in Niemann-Pick disease type C; clinical experience in four cases

In this case series, we demonstrate that Ursodeoxycholic acid (UDCA) improves liver dysfunction in Niemann-Pick type C (NPC) and may restore a suppressed cytochrome p450 system. NPC disease is a progressive neurodegenerative lysosomal storage disease caused by mutations in either the NPC1 or NPC2 ge...

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Detalles Bibliográficos
Autores principales:	Evans, William R.H., Nicoli, Elena-Raluca, Wang, Raymond Y., Movsesyan, Nina, Platt, Frances M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	F1000Research 2017
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5649119/ https://www.ncbi.nlm.nih.gov/pubmed/29119141 http://dx.doi.org/10.12688/wellcomeopenres.11854.1

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