disease 1,665 words KG: ent-dise-1b3c4df0 2026-03-22
kind:diseasesection:diseasesstate:published
Contents

Mitochondrial Membrane Protein-Associated Neurodegeneration (MPAN)

Disease Info
Lipid metabolismC19orf12 is co-regulated with genes involved in fatty acid biosynthesis and lipid homeostasis. Loss of C19orf12 leads to altered lipid composition of mitochondrial membranes
ER-mitochondria communicationDisruption of MAM integrity impairs calcium transfer between the ER and mitochondria, affecting cell survival signaling
[Autophagy](/entities/autophagy)C19orf12 deficiency leads to impaired autophagic flux, with accumulation of p62/SQSTM1 and ubiquitin-positive aggregates
Progressive spastic paraparesisLower limb spasticity with hyperreflexia and extensor plantar responses is often the earliest motor feature, distinguishing MPAN from other NBIA subtypes where extrapyramidal features predominate
DystoniaTypically generalized, progressive, and often severe; may be focal at onset before generalizing
ParkinsonismBradykinesia and rigidity develop with disease progression; tremor is less prominent than in Parkinson's disease
Cognitive declineProgressive intellectual deterioration, often with prominent executive dysfunction and eventually global dementia
Psychiatric featuresDepression, anxiety, emotional lability, impulsivity, and psychotic symptoms including visual hallucinations occur in up to 50% of patients (Hogarth et al., 2013)
Optic atrophyPresent in approximately 60% of patients; leads to progressive visual loss
Motor axonal neuropathyDistal weakness and muscle wasting due to motor neuron involvement; distinguishes MPAN from other NBIA subtypes
Dysarthria and dysphagiaProgressive bulbar dysfunction
Iron depositionHeavy iron accumulation in the globus pallidus (particularly the medial segment) and substantia nigra, with iron deposits in both neurons and glia
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (30)

SASP-Mediated Complement Cascade Amplification
Score: 0.70
Nutrient-Sensing Epigenetic Circuit Reactivation
Score: 0.67
Transcriptional Autophagy-Lysosome Coupling
Score: 0.66
Selective Acid Sphingomyelinase Modulation Therapy
Score: 0.65
CYP46A1 Overexpression Gene Therapy
Score: 0.63

Related Analyses (30)

TREM2 agonism vs antagonism in DAM microglia
neurodegeneration · failed
Selective vulnerability of entorhinal cortex layer II neuron
neurodegeneration · archived
4R-tau strain-specific spreading patterns in PSP vs CBD
neurodegeneration · archived
TDP-43 phase separation therapeutics for ALS-FTD
neurodegeneration · archived
Astrocyte reactivity subtypes in neurodegeneration
neurodegeneration · archived

Related Experiments (30)

ER-Golgi Secretory Pathway Dysfunction in PD - Experiment De
clinical · proposed · Score: 0.46
Proposed experiment from debate on Astrocytes adopt A1 (neur
falsification · proposed · Score: 0.46
s:** - Biochemical binding assays measuring PROTAC selectivi
falsification · proposed · Score: 0.46
Proposed experiment from debate on Epigenetic clocks and bio
falsification · proposed · Score: 0.46
Proposed experiment from debate on Microglia activate astroc
falsification · proposed · Score: 0.46

See Also (15)

Yoga Therapy for Neurodegeneration
therapeutic · Pages share 139 hypotheses
YAP/TEAD Pathway Modulators for Neurodegeneration
therapeutic · Pages share 139 hypotheses
Wnt Signaling Modulators for Neurodegeneration
therapeutic · Pages share 139 hypotheses
vitamin-d-therapy-neurodegeneration
therapeutic · Pages share 139 hypotheses
Vitamin B Complex Therapy for Neurodegeneration
therapeutic · Pages share 139 hypotheses