disease 1,242 words KG: ent-dise-9b7238bd 2026-03-20
kind:diseasesection:diseasesstate:published
Contents

Refsum Disease

Disease Info
PatternAutosomal recessive
Carrier frequencyApproximately 1 in 200 in general population
Phytanic acid accumulationLevels can reach 10-50 times normal (normal: < 30 μmol/L; patients: 300-3000 μmol/L)
Membrane incorporationPhytanic acid incorporates into neuronal and retinal cell membranes
Oxidative stressAccumulated phytanic acid promotes reactive oxygen species generation
Myelin dysfunctionAltered membrane properties affect nerve conduction<br>[^6]
Cardiac conduction abnormalitiesIncluding bundle branch block
Mild cognitive impairmentIn some patients
Skeletal abnormalitiesIncluding epiphyseal dysplasia
Typical onsetSecond to fourth decade
Range5-50 years
Retinitis pigmentosaUsually first manifestation
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph