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Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum)
2010-022046-25Phase 2
- Sponsor
- Department of Pediatric Gastroenterology, Amsterdam UMC
- Start
- Oct 2010
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, single-arm trial of cholic acid in Peroxisomal biogenesis disorder, Zellweger spectrum (PBD-ZSD), sponsored by Department of Pediatric Gastroenterology, Amsterdam UMC. Primary endpoint: Degree of suppression of endogenous bile acid synthesis.
02
Drugs
cholic acid
03
Conditions
Peroxisomal biogenesis disorder, Zellweger spectrum (PBD-ZSD)
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