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Assistance Publique - Hôpitaux de Paris
Treatment of bile acid synthesis disorders due to single enzyme defects
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Cholbam
(CHOLIC ACID)Orphan drugMirum 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...
William A Gahl, M.D.
National Human Genome Research Institute (NHGRI)
📍 Bethesda, Maryland
Wolfgang Köhler, MD
Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany
Amel Karaa, MD, M.D
Massachusetts General Hospital
📍 BOSTON, MA
Petros Kountouris, PhD
Cyprus Institute of Neurology and Genetics (CING)
David Goldfarb, MD
NYU Langone Health
Béatrice Gulbis, MD
Hôpital ERASME (ERASME)