disease 3,170 words KG: ent-dise-a10b7174 2026-03-24
kind:diseasesection:diseasesstate:publishedevidence:strong
Contents

Friedreich's Ataxia

Disease Info
Normal5-33 repeats
Intermediate (carrier)34-66 repeats
Pathological66-1700 repeats[@monros1999]
Reduced Fe-S cluster synthesisImpaired assembly of Fe-S clusters affects the function of multiple enzymes including aconitase, complexes I-III, and electron transfer flavoprotein[@muhlenhoff2018]
Iron accumulationMitochondrial iron overload with normal cytosolic iron levels leads to oxidative damage through Fenton chemistry[@bradley2000]
Energy failureReduced ATP production due to impaired oxidative phosphorylation[@rtig1997a]
Increased reactive oxygen species (ROS)Elevated ROS production causing lipid peroxidation, protein oxidation, and DNA damage[@emond2000]
Dorsal root ganglia (DRG)Most severely affected, with neurons loss, demyelination, and gliosis[@koeppen2009]
Cardiac muscleHypertrophic cardiomyopathy with fibrosis, leading to heart failure[@weidemann2019]
Cerebellar Purkinje cellsProgressive degeneration leading to ataxia[@koeppen2012]
Pancreatic β-cellsImpaired insulin secretion leading to diabetes mellitus[@cnop2008]
Skeletal muscleVariable involvement with reduced exercise tolerance[@lodi2002]
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph