Refsum Disease, Infantile (DBCOND0003498)

Identifiers

Synonyms

Infantile Refsum Disease / Infantile Refsum's Disease / Refsum's disease / Infantile Refsum's disease (disorder)

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT02171104	MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis	No drug interventions	treatment	2	recruiting
NCT00004442	Study of Bile Acids in Patients With Peroxisomal Disorders	Chenodeoxycholic acid Cholic Acid Ursodeoxycholic acid	treatment	Not Available	terminated
NCT00007020	Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid	Cholic Acid	treatment	3	completed