Study of Bile Acids in Patients With Peroxisomal Disorders
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Inherited Retinal Degenerative Disease Registry
Betaine and Peroxisome Biogenesis Disorders
† Study has passed its completion date and status has not been verified in more than two years.