Case Report: Ursodeoxycholic acid treatment in Niemann-Pick disease type C; clinical experience in four cases [version 1; referees: 2 approved]

Case Report: Ursodeoxycholic acid treatment in Niemann-Pick disease type C; clinical experience in four cases [version 1; referees: 2 approved]

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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Bibliographic Details
Main Authors: William R.H. Evans, Elena-Raluca Nicoli, Raymond Y. Wang, Nina Movsesyan, Frances M. Platt
Format: Article
Language:English
Published: Wellcome 2017-08-01
Series:Wellcome Open Research
Subjects:
Neurodevelopment
Pediatric Hepatology
Online Access:https://wellcomeopenresearch.org/articles/2-75/v1

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