disease 1,679 words KG: ent-dise-7542361c 2026-03-22
kind:diseasesection:diseasesstate:published
Contents

Canavan Disease

Disease Info
InheritanceAutosomal recessive; both parents must carry a pathogenic ASPA variant
PrognosisSignificantly better than infantile form; survival into adulthood is common
Ashkenazi Jewish populationCarrier frequency of approximately 1 in 37–40; disease incidence estimated at 1 in 6,400–13,500 births (Feigenbaum et al., 2004)
General populationCarrier frequency approximately 1 in 200; exact incidence unknown but significantly lower
Other populationsCases reported in diverse ethnic backgrounds including Saudi Arabian, Turkish, European, and Indian populations
p.Glu285Ala (E285A)Most common in Ashkenazi Jewish patients (~83% of alleles)
p.Tyr231Ter (Y231X)Second most common (~13% of Ashkenazi alleles)
p.Ala305Glu (A305E)Common in non-Jewish European patients
Early signs (1-3 months)Hypotonia, poor head control, feeding difficulties, macrocephaly
Motor regression (3-6 months)Loss of acquired motor milestones, progressive spasticity replacing hypotonia
Characteristic featuresSevere macrocephaly (head circumference >98th percentile), optic atrophy, seizures
MRI findingsDiffuse symmetric white matter involvement with elevated NAA on MR spectroscopy — pathognomonic for Canavan disease (Barkovich & Patay, 2019)
DatabasesOMIMOrphanetClinicalTrialsPubMed

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