Clinical Trial Update: Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid [Phase 3]

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Summary

Phase 3 trial update on compassionate use of cholic acid for infants with inborn errors of bile acid metabolism; study completed with results posted in 2020.

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Diseases / indications

• Infantile Refsum Disease
• Zellweger Syndrome

Drugs mentioned

• Cholic acid (cholic acid)

Snippet

Sponsor: Mirum Pharmaceuticals, Inc. | Conditions: Infantile Refsum's Disease, Zellweger Syndrome | Phase 3 | Status: COMPLETED | Results posted: 2020-07-15