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Hypercholanemia, familial

MONDO:0100327

Also known as: familial hypercholanemia, hypercholanemia, familial

1 clinical trial for this condition and its sub-types, 0 tagged with Hypercholanemia, familial itself.

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Where it sits in the disease tree

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Part of

↑ Inborn disorder of bile acid synthesis (12)

Sub-types of Hypercholanemia, familial

Hypercholanemia, familial 1 0 trials
Hypercholanemia, familial, 2 0 trials

Including sub-types (1) Tagged with Hypercholanemia, familial (0)

Not yet finished but already full! 1

Can a decade of real-world data refine treatment for rare bile acid disorders?

Knowledge-focused Ongoing

This study is a patient registry that will follow people of any age with bile acid synthesis disorders who are treated with Cholbam (cholic acid). The goal is to collect information over 10 years on how the drug performs in routine clinical care, including its safety, effectivene…

Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Knowledge-focused

Last updated Aug 02, 2026 00:00 UTC