Friedreich's Ataxia is the most common inherited ataxia in man. It is a mitochondrial disease
caused by severely reduced expression of the iron binding protein, frataxin. A large GAA
triplet expansion in the human FRDA gene encoding this protein inhibits expression of this
gene. It is inherited in an autosomal recessive pattern and typically diagnosed in childhood.
The primary symptoms include severe and progressive neuropathy, and a hypertrophic
cardiomyopathy that may cause death. The cardiomyopathy is difficult to treat and is …