Friedreich’s Ataxia

Friedreich’s ataxia (FA) is a rare, inherited neurodegenerative disease. It affects the nerves, spinal cord, and cerebellum (a part of the brain). It causes a slow, steady loss of muscle coordination and balance, called ataxia. It also affects speech and causes weakness and a loss of sensation. Over time, it can affect heart function.

FA occurs in about 1 in 50,000 people. It affects both males and females.

Friedreich’s ataxia causes

FA is inherited. That means it is passed from parents to children. It is caused by changes in a gene called FXN. This gene creates a protein called frataxin. Frataxin helps cells to produce energy. When a person has FA, their body produces less frataxin. Doctors can find this gene change with a genetic test.

To learn more about FA and current treatments, visit organizations dedicated to FA and rare diseases.

Friedreich’s ataxia care at St. Jude

St. Jude Children’s Research Hospital is working to deepen the understanding of rare, catastrophic genetic neurological disorders in children. Our aim is to transform the treatment landscape for these disorders. The Center for Experimental Neurotherapeutics (CENT) was established at St. Jude as part of the Pediatric Translational Neuroscience Initiative (PTNI) to focus on these neurological disorders.

Select children with FA who are accepted at St. Jude take part in clinical research or clinical trials. The aim of these studies is to help scientists learn more about the disorders and to develop potential treatments.

Friedreich’s ataxia clinical research

St. Jude offers clinical trials and cancer research studies for children, teens, and young adults for Friedreich’s ataxia. Learn more about clinical research at St. Jude.

Resources outside of St. Jude