disease 2,215 words KG: ent-dise-998d6a8a 2026-03-22
kind:diseasesection:diseasesstate:published
Contents

Pelizaeus-Merzbacher Disease (PMD)

Disease Info
PrevalenceApproximately 1 in 100,000 to 1 in 400,000 in the general population (Bonkowsky et al., 2010)
Typical OnsetFirst year
InheritanceX-linked recessive; predominantly affects males
Carrier femalesHeterozygous females are usually asymptomatic but may develop late-onset progressive neurological symptoms, particularly those carrying point mutations
Most common mutation typePLP1 gene duplications account for approximately 60-70% of PMD cases
Geographic distributionReported worldwide across all ethnic groups
PresentationSimilar to classic PMD but with prominent peripheral neuropathy
CourseMilder CNS involvement than classic PMD but with significant peripheral nerve demyelination
CognitionSeverely impaired
SurvivalTypically death from respiratory complications during childhood, though attentive care can extend survival into the third decade
GeneticsPLP1 deletions or null mutations
Distinctive featurePeripheral neuropathy differentiates this from other forms
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (5)

ACSL4-Driven Ferroptotic Priming in Disease-Associated Micro
Score: 0.66
Multi-Modal Stress Response Harmonization
Score: 0.60
Senescence-Induced Lipid Peroxidation Spreading
Score: 0.53
Extracellular Matrix Stiffness Modulation
Score: 0.43
Oligodendrocyte White Matter Vulnerability
Score: 0.40

Related Analyses (17)

TREM2 agonism vs antagonism in DAM microglia
neurodegeneration · failed
Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · archived
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · completed
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · archived
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived

Related Experiments (1)

ER-Golgi Secretory Pathway Dysfunction in PD - Experiment De
clinical · proposed · Score: 0.40