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...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wellcome
2017-08-01
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| Series: | Wellcome Open Research |
| Subjects: | |
| Online Access: | https://wellcomeopenresearch.org/articles/2-75/v1 |