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Updated: Jun 15 2021

Friedreich's Ataxia

Images
https://upload.orthobullets.com/topic/4087/images/scoli fre.jpg
https://upload.orthobullets.com/topic/4087/images/Colman block test - flexible hindfoot - courtesy Miller_moved.png
  • summary
    • Friedreich's Ataxia is an inherited disorder that leads to spinocerebellar degeneration due to an autosomal recessive mutation in the frataxin gene. The condition presents with ataxia, cardiomyopathy, motor weakness, cavovarus foot deformity, and scoliosis usually from age 7 to 25 year. 
    • Diagnosis is made with genetic testing to assess for the absence of the frataxin gene.
    • Treatment is initially nonoperative. Corrective surgery for symptomatic cavovarus foot deformity and progressive scoliosis may be indicated over time.
  • Epidemiology
    • Incidence
      • 1 in 50,000 births
    • Demographics 
      • onset usually between 7 and 25 years
        • age of onset related to number of GAA repeats
  • Etiology
    • Genetics
      • autosomal recessive
      • repeat mutation leads to lack of frataxin gene
        • frataxin is a mitochondrial protein involved in iron metabolism and oxidative stress
        • mutation is GAA repeat at 9q13
    • Associated conditions
      • pes cavovarus foot
      • scoliosis
        • predictors of progression
          • onset of disease is less than 10 years of age
          • onset of scoliosis is before 15 years of age
      • cardiomyopathy
        • cardiology evaluation before surgery
        • antioxidants (Coenzyme Q) have been shown to decrease rate of cardiac deterioration but have no effect on ataxia
  • Presentation
    • Symptoms
      • ataxia
        • staggering wide based gait (spinocerebellar)
    • Physical exam
      • classic triad
        • ataxia
        • areflexia
        • positive plantar response
      • weakness
      • nystagmus
      • cavovarus foot
        • very high arch
        • rigid deformity
        • associated claw toes
      • scoliosis
  • Imaging
    • Radiographs
      • recommended views
        • standing scoliosis series
        • AP and lateral of foot if pes cavovarus present
  • Studies
    • EMG
      • shows defects in motor and sensory with an increase in polyphasic potentials
      • nerve conduction velocities are decreased in upper extremities
  • Treatment
    • Cavovarus foot
      • nonoperative
        • observation
          • indications
            • only indicated in nonambulatory patient
            • deformity is rigid and progressive and resistant to bracing and stretching as treatment
      • operative
        • plantar release, transfers, +/- metatarsal and calcaneal osteotomy
          • indications
            • early disease in ambulatory patient
        • triple arthrodesis
          • indications
            • late disease in nonambulatory patients
    • Scoliosis
      • nonoperative
        • observation
          • indications
            • curves < 40 degree without predictors of progression (see above)
      • operative
        • PSF and instrumentation
          • indications
            • curve > 60 degrees
            • rapid progression with positive predictors of progression
              • onset of disease is less than 10 years of age
              • onset of scoliosis is before 15 years of age
            • usually does not need to be extended to pelvis
  • Prognosis
    • Usually wheelchair bound by age 30
    • Usually die by age 50 from cardiomyopathy
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