disease 2,858 words KG: ent-dise-42c06396 2026-03-21
kind:diseasesection:diseasesstate:published
Contents

Charcot-Marie-Tooth Disease

Disease Info
PrognosisVariable, but generally more progressive than dominant forms; many patients require wheelchair by the second or third decade
Age of onsetUsually in the first decade of life; many children present with delayed motor milestones or difficulty walking
SeverityOften more disabling than CMT1, with earlier wheelchair dependence in some subtypes
Geographic distributionMore prevalent in populations with higher rates of consanguinity (Mediterranean, Middle Eastern, North African populations) due to recessive inheritance
CMT4A (GDAP1)Ganglioside-induced differentiation-associated protein 1 mutations; both demyelinating and axonal forms; associated with vocal cord paresis
CMT4B1/B2 (MTMR2/MTMR13)Myotubularin-related proteins; distinctive "focally folded" myelin sheaths on nerve biopsy
CMT4C (SH3TC2)SH3 domain and tetratricopeptide repeats 2; most common CMT4 subtype; scoliosis is a prominent feature
CMT4F (PRX)Periaxin; severe sensory loss with sensory ataxia
CMT4J (FIG4)Phosphoinositide phosphatase; rapidly progressive course
Clinical featuresProminent scoliosis, foot deformities, distal limb weakness, and sensory loss; cranial nerve involvement may occur in some subtypes
NCV valuesIntermediate range (25–45 m/s in males), which can complicate classification. Females typically show higher NCV values than affected males
Other CMTX typesCMTX2–CMTX6 are rare subtypes caused by mutations in AIFM1, GJB3, and other X-linked genes
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (12)

Bacterial Enzyme-Mediated Dopamine Precursor Synthesis
Score: 0.36
Senescent Cell Mitochondrial DNA Release
Score: 0.54
Near-infrared light therapy stimulates COX4-dependent mitoch
Score: 0.51
TFAM overexpression creates mitochondrial donor-recipient gr
Score: 0.47
Mitochondrial Transfer Pathway Enhancement
Score: 0.44

Related Analyses (16)

What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · archived
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
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived

Related Experiments (2)

Macroautophagy Dysfunction in PD - Experiment Design
clinical · proposed · Score: 0.40
Retrospective Clinical Survey of Hereditary Neuromuscular Di
clinical · proposed · Score: 0.90