Congenital intestinal disease due to an enzymatic defect Advocacy Hub | UniteRare

At a Glance

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1FDA treatments

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No FDA or trial events recorded yet for Congenital intestinal disease due to an enzymatic defect.

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FINANCIAL LANDSCAPE SUMMARY

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Approved Treatments

1 FDA-approved

Cholbam

(CHOLIC ACID)Orphan drugstandard

Mirum Pharmaceuticals Inc.

Bile Acid [EPC]

12.1 Mechanism of Action Cholic acid is a primary bile acid synthesized from cholesterol in the liver. In bile acid synthesis disorders due to SEDs in...

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Clinical Trial Landscape

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Recent News & Research

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