A New Chapter in Friedreich’s ataxia (FA)

What’s missing in Friedreich’s ataxia (FA) treatment?

In the last few years, there have been advances in the management of FA. However, it’s time to turn the page and focus on the root cause—frataxin deficiency—and the potential of increasing frataxin levels as a therapeutic target.

According to 50 physicians who treat FA, 94 percent agree there are still patient needs that are not being met in FA treatment.

Understanding FA

FA is a rare, progressive disease that affects multiple parts of the body.²

Signs and symptoms of FA include³

Illustration of a body with the signs and symptoms of Friedreich’s ataxia labeled: fatigue, vision and hearing impairment, slurred speech, heart conditions, scoliosis, diabetes, loss of coordinated movement, and difficulty with balance.

Talk to your doctor if you are experiencing these symptoms.

How do people get FA?

How do people get Friedreich’s ataxia (FA)?

FA is an inherited disorder where the frataxin gene is mutated, affecting the production of an important protein called frataxin.⁴

How many
people have FA?

How many people have Friedreich’s ataxia (FA)?

FA affects 1 in every 40,000 people globally and approximately 5000 in the US.^5-7

When do people
start experiencing
FA symptoms?

When do people start experiencing Friedreich's ataxia (FA) symptoms?

Symptoms of FA typically appear between the ages of 5 and 20 and sometimes can present in individuals as late as in their 60s.²

How is FA diagnosed?

How is Friedreich’s ataxia (FA) diagnosed?

Your doctor can confirm an FA diagnosis with genetic testing.⁸

What does
an FA care team
look like?

What does a Friedreich’s ataxia (FA) care team look like?

There is a wide array of doctors that are involved in the management of FA, including primary care providers; neurologists; cardiologists; physical, occupational, and speech therapists; endocrinologists; orthopedic surgeons; and mental health professionals.⁹

How is FA
evaluated?

How is Friedreich’s ataxia evaluated?

Your doctor may conduct a series of assessments to check your balance, sensations, reflexes, and more.¹⁰

Frataxin matters in Friedreich's ataxia (FA), and there's still more to understand

FA is caused by a deficiency in the protein frataxin.²

(Left image) A photo of cells circulating in the body against a purple-blue background. (Right image) An illustration of where frataxin is located in the mitochondria of a cell.

Frataxin is a key protein that supports the production of energy for multiple systems in the body, including the nervous system and heart.¹⁰ With decreased frataxin, the cells in the body are less able to make the energy the body needs to function.⁵

People with FA have significantly reduced levels of frataxin compared to healthy individuals. Lower levels of frataxin typically correlate with earlier disease onset and more severe symptoms.²

A new chapter in Friedreich’s ataxia (FA) is unfolding

Research has revealed the potential of increasing frataxin levels through therapeutic approaches such as frataxin replacement therapy, gene therapy, and small molecule medicine—welcoming a new chapter in FA.

A young man with Friedreich's ataxia riding in a motorized chair on a stone path in a lush, green forest.

Stay tuned for the next chapter

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References:

Larimar Therapeutics–sponsored survey, data on file.
Keita M, McIntyre K, Rodden LN, Schadt K, Lynch DR. Friedreich ataxia: clinical features and new developments. Neurodegener Dis Manag. 2022;12(5):267-283.
Alliance for Patient Access and Friedreich’s Ataxia Research Alliance. Friedreich’s ataxia: a rare condition facing common access challenges. Patient brochure. Accessed August 18, 2025. https://allianceforpatientaccess.org/wp-content/uploads/2025/02/AFPA-RareWG-FAPaper-Feb2025.pdf.
Ercanbrack WS, Ramirez M, Dungan A, Gaul E, Ercanbrack SJ, Wingert RA. Frataxin deficiency and the pathology of Friedreich’s ataxia across tissues. Tissue Barriers. Published online February 21, 2025. doi:10.1080/21688370.2025.2462357.
Friedreich’s Ataxia Research Alliance. What is Friedreich’s ataxia? Accessed August 19, 2025. https://www.curefa.org/understanding-fa/what-is-friedreichs-ataxia/.
Williams CT, De Jesus O. Friedreich ataxia. In: StatPearls. Updated August 23, 2023. Accessed September 11, 2025. https://www.ncbi.nlm.nih.gov/books/NBK563199/.
Lopez M. Friedreich ataxia (FA). Rare disease advisor. Updated January 16, 2023. Accessed September 11, 2025. https://www.rarediseaseadvisor.com/disease-info-pages/friedreich-ataxia-epidemiology/.
Indelicato E, Delatycki MB, Farmer J, et al. A global perspective on research advances and future challenges in Friedreich ataxia. Nat Rev Neurol. 2005;21(4):204-215.
Friedreich’s Ataxia Research Alliance. Assemble a care team. Understanding FA. Accessed August 18, 2025. https://www.curefa.org/understanding-fa/managing-fa/#care.
National Institute of Neurological Disorders and Stroke. Friedreich ataxia. National Institutes of Health. Updated June 9, 2025. Accessed August 18, 2025. https://www.ninds.nih.gov/health-information/disorders/friedreich-ataxia.