disease 2,454 words KG: ent-dise-800afaa9 2026-03-24
kind:diseasesection:diseasesstate:published
Contents

Wolfram Syndrome

Disease Info
PrevalenceEstimated at 1 in 770,000 in the United Kingdom; 1 in 54,478 in a Sicilian district of Italy, reflecting higher consanguinity rates (Barrett et al., 1995; Lombardo et al., 2014)
InheritanceAutosomal recessive for both types, though heterozygous WFS1 variants can cause dominantly inherited sensorineural hearing loss and diabetes
PrognosisMedian survival approximately 30 years (range 25-49 years)
Carrier frequencyApproximately 1 in 354 in the UK general population
Higher prevalenceIn populations with high rates of consanguinity (Middle East, North Africa, South Asia)
Type distributionWS1 (WFS1 mutations) accounts for ~90% of cases; WS2 (CISD2 mutations) is extremely rare, with cases reported in only a few families worldwide
Sex distributionAffects males and females equally
GeneCISD2 (chromosome 4q24)
Core featuresDiabetes mellitus, optic atrophy, sensorineural deafness
NeurodegenerationSimilar progressive neurological decline
UrologicalNeurogenic bladder, hydroureteronephrosis
PsychiatricDepression, anxiety, psychosis (present in ~60% of patients)
DatabasesOMIMOrphanetClinicalTrialsPubMed

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