Journal of pediatric gastroenterology and nutrition
Baseline analysis of patient reported outcomes in the progressive familial intrahepatic cholestasis patient registry.
Ryan T Fischer, Emily Ventura, James E Squires, Walter Perez, Akihiro Asai, Melissa Kochanowsky, Benjamin L Shneider, Gitta H Lubke
Published: 202610.1002/jpn3.70366
Abstract
OBJECTIVES: Progressive familial intrahepatic cholestasis (PFIC) is an overarching term for rare monogenic defects that result in cholestatic liver disease. Larger consortia-based registries have begun to address the challenges of PFIC as a rare dise…
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