Publications by authors named "Vera F Hupertz"
Article Synopsis
- Progressive familial intrahepatic cholestasis (PFIC) is an autosomal recessive disorder mainly caused by BSEP deficiency, leading to issues like bile formation disruption and severe itching (pruritus).!* -
- The MARCH-PFIC study was a phase 3, double-blind, placebo-controlled trial involving participants aged 1-17 from 29 centers across 16 countries, focusing on the effects of the drug maralixibat on different types of PFIC.!* -
- The study aimed to measure changes in pruritus severity and serum bile acid levels over 26 weeks, specifically looking at outcomes in cohorts with varying forms of PFIC, comparing those treated with maralixibat