Friedreich ataxia (FRDA) is a recessive disorder predominantly beginning in childhood or adolescence that causes progressive ataxia and dysarthria along with loss of sensation and coordination.1 Cardiomyopathy, scoliosis, diabetes, sensorineural hearing loss, and optic neuropathy are further manifestation of the disease. FRDA is caused by biallelic mutations in the FXN gene, which codes for the protein frataxin. The most common mutation is a biallelic expansion of a naturally occurring guanine-adenine-adenine (GAA) repeat in intron 1 (96%), while the remaining 4% of individuals carry 1 expansion along with a point mutation or a deletion. All disease-causing genotypes decrease, but not totally deplete, functional frataxin. FRDA is therefore primarily a disease of frataxin deficiency. Frataxin carries a mitochondrial targeting sequence, and its relative absence leads to the dysfunction of multiple mitochondrial processes. From a mechanistic point of view, all disease manifestations in FRDA reflect dysfunctions downstream of frataxin deficiency and reflect a primary mitochondrial pathology.