← All indications
Refsum Disease, Infantile
An early onset form of phytanic acid storage disease with clinical and biochemical signs different from those of REFSUM DISEASE. Features include MENTAL RETARDATION; SENSORINEURAL HEARING LOSS; OSTEOPOROSIS; and severe liver damage. It can be caused by mutation in a number of genes encoding proteins involving in the biogenesis or assembly of PEROXISOMES.
Drugs by status
Phase 3 trials (1)
Phase 2 trials (3)
Other trials (2)
Organizations
Organization Involved with Phase 3 Indications (2)
Organization Involved with Phase 2 Indications (1)