Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Zellweger syndrome

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Hydroxychloroquine Administration for Reduction of Pexophagy Study of Bile Acids in Patients With Peroxisomal Disorders Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid