Managing FA - Friedreich's Ataxia Research Alliance

Resources for Managing FA

All individuals with FA experience ataxia and neurologic symptoms but the severity and progression of these symptoms differ person by person.

Other symptoms of FA, like cardiomyopathy, diabetes, and scoliosis, also vary between individuals and are not always present. It’s important to monitor these symptoms so they can be promptly managed if or when they appear. The resources and suggestions below are intended to help you no matter where you are in your FA journey, from newly diagnosed to living with FA for many years. As your symptoms of FA change over time, your FA care and management will also evolve.

The resources below are meant to serve as educational starting points for your care. All healthcare decisions should be made with the support of a healthcare professional and with your unique symptoms, health issues outside of FA, and health history in mind.

Clinical Management Guidelines for Friedreich’s Ataxia

FA experts from around the world have written FA Clinical Management Guidelines to help you and your care team make decisions about your care that work best for you.

The website is organized into topic chapters with lay summaries, best practice statements, links to the evidence, and recommendations for each topic. Some of the topics include: neurological components, mental health, emergency medicine, fatigue, orthopedic issues, endocrine & metabolism, the heart and cardiovascular system.

Access the Guidelines

Photo of a girl taking a peg board test.

Assemble a Care Team

Most people with FA receive care from a variety of specialists to manage and monitor their symptoms. Everyone with FA experiences different types and degrees of symptoms, so each person’s care team will look different. Below, you can see some common members of the FA care team. Neurologists are often at the center of an FA care team. One way to find a neurologist and other healthcare professionals knowledgeable in FA is to visit an FA Global Clinical Consortium site. Some people with FA find it helpful to visit an FA expert once a year to receive recommendations and advice they can take back to their local doctors to help guide their care.

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Care Team Members

Neurologists assess and manage symptoms related to ataxia (balance and coordination), pain, spasticity, sensory loss and muscle function.

Cardiologists monitor the heart for signs of cardiomyopathy (thickening of the heart muscle) and arrhythmias (abnormal heart rhythm) and treat cardiac symptoms.

Physical and occupational therapists help people who have FA to build strength and learn how to modify daily tasks. Speech therapists can help manage symptoms of slurred speech and difficulty swallowing.

Even if you are seeing countless specialists for FA, having a primary care provider ensures you have someone local to go to for urgent situations, illnesses, and injuries.

Endocrinologists can help manage diabetes, growth differences, and low bone density.

Orthopedists can help manage scoliosis (curving of the spine) and pes cavus (high arched feet).

Many people with FA experience mental health difficulties including anxiety and depression. Therapists, psychologists, and psychiatrists can help manage these issues.

Other specialists someone with FA might see to manage symptoms include audiologists, ophthalmologists, dietitians, sleep specialists or pulmonologists, and geneticists or genetic counselors.

Sometimes, it may be up to you to explain FA to a new doctor who has never heard of it. When it comes to you or your child’s daily experience with FA, you are the expert in the room.

Navigating appointments with doctors who are unfamiliar with FA

FA is a rare disease, so it is possible that physicians and healthcare professionals near you might not know a lot about managing FA symptoms. Don’t be afraid to speak up in these situations and share information you have learned on this page and provide any doctors unfamiliar with FA with the clinical management guidelines.

As you work to build an FA care team, it may take some time to find doctors and clinicians who are the right fit for your team. Even if a specialist is unfamiliar with FA, what’s important is their willingness to learn and collaborate with you and other members of your care team. It is common for individuals with FA to see specialists or experts in FA once a year at a hospital or clinic far away from home and then have local physicians who manage things based on the FA expert recommendations.

Treatments for Friedreich’s Ataxia

The first treatment for FA, SKYCLARYS™, was approved in the United States in 2023 and in the European Union in 2024 for the treatment of people with FA, age of 16 and older. SKYCLARYS is a once daily oral medication that has been shown to slow progression of FA symptoms in a clinical trial. More information about SKYCLARYS is available on the Approved Treatment page.

Other therapeutics are needed to slow, stop, reverse, and cure FA. FARA believes in a cocktail approach – a combination of two or more therapies. Several potential future treatments for FA are currently being investigated. You can find information about these potential treatments on the Drug Development Pipeline page.

Approved treatment Pipeline

Doctors Answer Frequently Asked Questions about FA

Doctors David Lynch (neurology), Shana McCormack (endocrinology), and Kimberly Lin (cardiology) answered frequently asked questions about FA clinical management at the FARA and Children’s Hospital of Philadelphia annual FA Symposium in 2023. Watch the full video or click the links below to be directed to answers for specific questions.

Everyone with FA has a unique set of symptoms, and many people may have other health factors that influence their care outside of FA symptoms. It’s important to work with your care team to make medical decisions that make sense in the context of your own health.

Watch the full video