familial isolated deficiency of vitamin E

MeSH: C535393ORPHA: 96

Overview

rare autosomal recessive neurodegenerative disease. Symptoms are similar to those of Friedreich ataxia

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 familial isolated deficiency of vitamin E, sourced from HPO and Orphanet clinical annotations.

AtaxiaAreflexiaMuscle weaknessBabinski signAbnormal pyramidal signPeripheral neuropathyDecreased circulating vitamin E concentrationNystagmusNyctalopiaSensory neuropathyDysarthriaCerebellar atrophyGait disturbanceDysmetriaPes cavusDysdiadochokinesisAbnormal speech patternClumsinessHead titubationScoliosisImpaired proprioceptionUrinary urgencyVisual impairmentAbnormality of visual evoked potentialsDiabetes mellitusMental deteriorationHypertoniaDystoniaTremorHypertrophic cardiomyopathyDevelopmental regressionPositive Romberg signSkeletal muscle atrophyHemiplegia/hemiparesisAbnormal retinal pigmentationArrhythmiaPigmentary retinopathy

Classification & Codes

MeSH Code

C535393

Orphanet Code

ORPHA:96
familial isolated deficiency of vitamin E
MeSHC535393
OrphanetORPHA:96
Treatments0 drug(s)
Symptoms on record37 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO