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PFIC2

Clinical trials for PFIC2 explained in plain language.

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  • Hope for kids with rare liver disease: new drug targets debilitating itching and liver damage

    Disease control Completed

    This study tested whether a drug called A4250 could help children with rare, progressive liver diseases known as PFIC1 and PFIC2. The main goals were to see if the drug could lower harmful bile acids in the blood and reduce the severe, life-altering itching these children experie…

    Matched conditions: PFIC2

    Phase: PHASE3 • Sponsor: Albireo • Aim: Disease control

    Last updated Mar 25, 2026 14:08 UTC

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