Hereditary Ataxia, Freidrich's Ataxia



Friedrich's Ataxia:


Presentation:

  1. Cerebellar Syndrome= Ataxia/broad based gait+horozontal nystagmus+staccato speech+intention tremor
  2. Dorsal Column= Loss of vibration and proprioception+ Romberg test positive
  3. Pyramidal lesion= Spastic paraparesis/Upper limb exptensor weaker, so in flexed position+ Lower limb fexor weaker, so in extended position, Extensor Plantar
  4. Peripheral neuropathy= Absent ankle jerk, long term= Pes cavus=hereditary
  5. Others: 
  • Diabetes Mellitus
  • Optic atrophy(bilateral)
  • HOCM= Sacr mark=ICD
  • High arched Palate =  D/D MArfan's/Homocystinuria/Turner's/tuberous sclerosis = Ataxia absent 
  • peScavus+kyphoSCLOLIOSIS+Sensoryneural hearing loss
  • Family history positive

Age of presentation:

  • 10-15 years/below 30 years
  • some late onset milder form is now detected, due to genetic test facility

Diagnosis:

  • Confirmed by genetic test = GAA repeat ( upto 50 is normal, In FA, it can be >200, or even >1000) 
  • Autosomal Recessive+GAA repeat+ X25 gene+ Chromosome 9+Frataxin + No anticipation
  • Frataxin= iron regulation in mitochondria, If frataxin absent, iron deposition in mitochondria

  • ECG= T wave inversion+ LVH
  • MRI= Shrinkage of cervical cord
  • Nerve Conduction Study= Sensory amplitude low/absent, motor- normal

D/D:

  •  Hereditary Sensori-motor Neuropathy= Ataxia absent
  • Tabes dorsalis= Argyll robertson pupil present

Treatment:

  • Multi Disciplinary aprroach:
  • Neurologist+Cerdiologist+Endocrinologist+ Physiotherapist+Occupational therapist+ Orthopedic surgeon+genetic counselor

  • 2E+2A+2O
  • Education= genetic advice
  • Exercise and physiotherapy
  • Aid=walking+visual+hearing
  • Ankle support=orthoses
  • Occupational therapy
  • Orthopedic surgery= to correct pes cavus+ kyphoscoliosis

  • Endocrinology= DM management
  • Cardiology= HOCM management

Prognosis:

  • Progress variable
  • Unable to walk/ wheel chair bound: after 15 years of onset
  • Leg weakness→Wheel chair bound→Arm weakness

Causes of absent ankle jerk and extesor plantar:

  • CVD with peripheral neuropathy( DM)
  • Cervical myelopathy with peripheral neuropathy
  • Cervical and lumbar spondylosis
  • Conus medularis lesion
  • Freidrich's Ataxia
  • Subacute combined degeneration of spinal cord
  • Taboparesis
  • Motor neuron disease

Early onset recessive ataxia :


  • Freidrich's ataxia
  • Ataxic Telangiectasia
  • Abetalipoproteinemia
  • Refsum's disease






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