Peroxisomal Disorders Medication

Updated: Jul 29, 2020
  • Author: Hoda Z Abdel-Hamid, MD; Chief Editor: Stephen L Nelson, Jr, MD, PhD, FAACPDM, FAAN, FAAP, FANA  more...
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Gastrointestinal Agents, Other

Class Summary

Deficiency of primary bile acids leads to unregulated accumulation of intermediate bile acids and cholestasis. Primary bile acid replacement therapy has been shown to improve liver function and weight gain. [9, 10]

Cholic acid (Cholbam)

Endogenous bile acids, including cholic acid, enhance bile flow and provide the physiologic feedback inhibition of bile acid synthesis. Cholic acid is indicated for adjunctive treatment of peroxisomal disorders (PDs), including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.