Mirum Pharmaceuticals, Inc.
Clinical trials sponsored by Mirum Pharmaceuticals, Inc., explained in plain language.
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Hope for rare liver disease: drug aims to ease severe itching in children
Disease control CompletedThis study tests the long-term safety of maralixibat, an oral medication, in children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes intense itching. Participants who completed a prior study will receive maralixibat twice daily. Resear…
Phase: PHASE3 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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New drug shows promise for babies with rare liver disorders
Disease control CompletedThis study tested the safety of a drug called maralixibat in 27 infants under 12 months old with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC), two rare liver diseases that cause bile buildup. The drug is given as an oral solution and aims to reduce bi…
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Liver drug maralixibat tested for Long-Term safety in rare disease patients
Disease control CompletedThis study looked at the long-term safety of a drug called maralixibat in 52 people with cholestatic liver disease, including Alagille Syndrome and other rare conditions. Participants had already taken maralixibat in an earlier study. Researchers tracked side effects and how well…
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC