Friedrich’s Ataxia

Friedrich’s Ataxia


FRIEDREICH’S ATAXIA

Etiology:

  • Autosomal Recessive disorder,
  • Most common form of Inherited Ataxiae
The classic form of Friedreich’s ataxia has been mapped to 9q13-q21.1, and the mutant gene, frataxin, contains expanded GAA triplet repeats in the first intron. There is homozygosity for expanded GAA repeats in >95% of patients. 
Normal persons have 7–22 GAA repeats, and patients have 200–900 GAA repeats.
 

Characteristic Diagnostic features:

  • Chronic Slowly progressive cerebella,- ataxia
  • Absent lower limb deep tendon reflexes with an extensor plantar response
  • Associated features of
  •  Cardiomyopathy
  • Diabetes
  •  Spine or foot deformity
 
Clinical and Histologic manifestations:
 
  • The primary sites of pathology: spinal cord, dorsal root ganglion cells, and the peripheral nerves
  • The cerebral cortex is histologically normal except for loss of Betz cells in the precentral gyri
  • Chronic progressive wide based ataxia, Progressive staggering gait
  • Frequent falls, truncal titubation
  • Loss of fast saccadic eye movements -Nystagmus, Dysarthria, Dysmetria Titubation
  • The median age of death is 35 years
  • Women have a significantly better prognosis than men

Other Characteristic Manifestation

  1. Orthopaedic
  2. Kyphoscoliosis (spine defbrinity) Pescavus (foot deformity)
  3. Cardiac
  4. Cardiomyopathy (90%)
  5. Endocrine
  6. Diabetes (20%)
  7. Eye
  8. Optic and Retinal Atrophy
  9. Psychiatric
  10. Dementia
Exam Question

FRIEDREICH’S ATAXIA

Etiology:

The classic form of Friedreich’s ataxia has been mapped to 9q13-q21.1, and the mutant gene, frataxin, contains expanded GAA triplet repeats in the first intron. There is homozygosity for expanded GAA repeats in >95% of patients.

Characteristic Diagnostic features:

  • Chronic Slowly progressive cerebella,- ataxia
  • Absent lower limb deep tendon reflexes with an extensor plantar response
  • Associated features of
  • Cardiomyopathy
  • Diabetes
  •  Spine or foot deformity
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