disease 774 words KG: ent-dise-2e09443f 2026-03-29
kind:diseasesection:diseasestopic:aoa2
Contents

Ataxia with Oculomotor Apraxia Type 2 (AOA2) Genetic Variants

Disease Info
Typical OnsetTypically adolescence (mean age 12-15 years)
Nonsense mutations~40% — premature stop codons, typically severe phenotype
Missense mutations~35% — may retain partial function, variable severity
Frameshift mutations~15% — insertions/deletions causing truncations
Splice site mutations~10% — abnormal mRNA processing
Two truncating mutationsEarlier onset (mean 7 years), more rapid progression
Missense compound heterozygousLater onset, slower progression
Residual senataxin activityCorrelates with milder phenotype
Progressive cerebellar ataxiaGait instability, limb incoordination, dysarthria (scanning speech)
Oculomotor apraxiaDifficulty initiating voluntary eye movements, especially horizontal gaze — patients use head thrusts to compensate
Elevated AFPPersistent elevation of serum alpha-fetoprotein is a key diagnostic marker (normal in A-T)
Sensorimotor neuropathyDistal weakness, reduced or absent deep tendon reflexes
DatabasesOMIMOrphanetClinicalTrialsPubMed

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