disease 989 words KG: ent-dise-800c83d9 2026-03-24
kind:diseasesection:diseasesstate:published
Contents

Metachromatic Leukodystrophy ([MLD](/diseases/metachromatic-[leukodystrophy](/diseases/leukodystrophy)))

Disease Info
Prevalence1 in 40,000 - 1 in 160,000 (varies by population)
Carrier Frequency~1 in 40-80 in general population
Founder MutationsCommon in Amish, Mennonite, Portuguese populations
[ARSA](/genes/arsa)-PseudodeficiencyCommon variant with reduced but not absent enzyme activity
No Clinical SymptomsUsually benign; can complicate diagnosis
Missense MutationsMost common; genotype-phenotype correlation variable
Nonsense MutationsOften cause severe (infantile) form
Splice Site MutationsCan cause frameshift and premature termination
Large DeletionsLess common, severe phenotype
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph