Friedreich's ataxia is a debilitating, life-shortening, degenerative, neuro-muscular disorder. There are approximately 5,000 patients in the US and 15,000 globally.  It is a single gene defect disorder that prevents the production of a protein frataxin (FXN).  Frataxin is a vital protein whose function is helping the mitochondria produce energy.

Neurological cells degenerate in FA patients over time. This results in:

• Loss of feeling and control of arms and legs (ataxia)
• Aggressive scoliosis
• Hypertrophic cardiomyopathy and arrhythmia
• Fatigue, energy deprivation, and muscle loss
• Diabetes
• Vision impairment, hearing loss and slurred speech