adult Refsum disease

MeSH: D012035ORPHA: 773

Overview

Human disease

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with adult Refsum disease, sourced from HPO and Orphanet clinical annotations.

Renal insufficiencyAnosmiaAbnormality of the eyeRetinopathyAbnormality of eye movementAbnormality of visionVisual impairmentPtosisCataractProgressive visual lossMicrophthalmiaNystagmusNyctalopiaDry skinAtaxiaCardiomyopathySplenomegalySensorineural hearing impairmentMiosisHypotoniaAbnormal foot morphologyPes cavusHammertoeAbnormality of metabolism/homeostasisRespiratory insufficiencyNail dysplasiaDevelopmental regressionSkeletal dysplasiaSkeletal muscle atrophyHemiplegia/hemiparesisAbnormal epiphysis morphologyAbnormal pyramidal signAbnormal retinal pigmentationIchthyosisPeripheral neuropathyShort metacarpalSevere intellectual disabilityHeart block

Classification & Codes

MeSH Code

D012035

Orphanet Code

ORPHA:773
adult Refsum disease
MeSHD012035
OrphanetORPHA:773
Treatments0 drug(s)
Symptoms on record38 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO