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PubMed This is a summary of 21 peer-reviewed journal articles Updated
Neurology

Treatment & Care Team: Managing Friedreich Ataxia

At a Glance

The first FDA-approved treatment for Friedreich Ataxia is omaveloxolone (Skyclarys), which helps slow disease progression. Because FRDA affects multiple organs, patients require a multidisciplinary care team led by a neurologist, including cardiologists, orthopedists, and specialized therapists.

Managing Friedreich Ataxia (FRDA) has transitioned from purely treating symptoms to a more proactive approach that integrates the first approved disease-modifying therapy with a specialized, multisystem care team [1][2]. Because FRDA affects multiple organs, your medical team should work like a coordinated network rather than individual doctors in isolation. Organizations like FARA (Friedreich’s Ataxia Research Alliance) can help connect you with specialized care centers.

The First Approved Therapy: Omaveloxolone

Omaveloxolone (brand name Skyclarys) is the first FDA-approved pharmacological treatment specifically for Friedreich Ataxia [3].

  • Who it is for: It is currently approved for adults and adolescents aged 16 and older [3][4]. Pediatric clinical trials are actively ongoing for patients younger than 16.
  • How it works: It activates the Nrf2 pathway, a biological switch that helps restore mitochondrial function and reduces the cellular inflammation and stress caused by low frataxin levels [5][6].
  • What it does: In clinical trials, it has been shown to improve neurological function and slow the progression of the disease as measured by the mFARS (modified Friedreich Ataxia Rating Scale) [7][8].
  • Safety Warning: While generally well-tolerated, omaveloxolone carries a prominent risk of fluid retention, which can exacerbate heart failure [9][10]. Because FRDA patients are already at risk for cardiomyopathy, you should immediately report any swelling, shortness of breath, or sudden weight gain to your doctor. Doctors will monitor liver enzymes and heart-related markers before and during treatment.

Building Your Multidisciplinary Team

Because FRDA is a multisystem disorder, you will need several specialists to monitor different aspects of your health [2][11]:

  • Movement Disorder Neurologist: This is typically the “captain” of your care team. They specialize in ataxia and use standardized scales (like mFARS) to track your progression and manage neurological symptoms [2][12].
  • Cardiologist: Heart health is critical. A cardiologist will perform regular EKGs and echocardiograms to monitor for cardiomyopathy (thickening of the heart muscle) or arrhythmias [13][14].
  • Orthopedist: They monitor the spine for scoliosis and the feet for pes cavus (high arches), recommending braces or surgery if necessary [15][16].
  • Endocrinologist: They monitor for diabetes mellitus and manage blood sugar levels if the pancreas is affected [11].
  • Physical and Occupational Therapists (PT/OT): PT focuses on maintaining mobility and balance, while OT helps with tools for independence in daily activities and energy conservation [17][18].
  • Speech-Language Pathologist (SLP): They help with communication strategies for dysarthria (speech changes) and perform swallow studies to ensure you are eating safely and not at risk for aspiration [19].

Evaluating Your Care

When choosing specialists, it is helpful to look for those associated with the Friedreich’s Ataxia Global Clinical Consortium or a “Center of Excellence” [20]. These centers participate in the Collaborative Clinical Research Network (CCRN), ensuring they are at the forefront of new treatments and clinical trials.

Standard Care Strategies

Standard management involves proactive screening and support:

  • Mobility Support: Comprehensive rehabilitation programs are essential for maintaining independence for as long as possible [17].
  • Surgical Intervention: For advanced scoliosis, a posterior spinal fusion may be considered to prevent further curvature, though these procedures require specialized care due to the multisystem nature of FRDA [16].
  • Heart Monitoring: Routine cardiac evaluations are the standard of care because heart complications remain a primary concern in FRDA management [14][21].

Common questions in this guide

What is the first approved treatment for Friedreich Ataxia?
Omaveloxolone, sold under the brand name Skyclarys, is the first FDA-approved medication for Friedreich Ataxia. It works by activating a cellular pathway that helps restore cell function and reduces the cellular inflammation caused by low frataxin levels.
Which doctors should be on my Friedreich Ataxia care team?
Because FRDA affects multiple body systems, your care team should include a movement disorder neurologist, a cardiologist, an orthopedist, and an endocrinologist. Physical, occupational, and speech therapists are also essential for maintaining daily mobility and communication.
Why do I need to see a cardiologist if I have Friedreich Ataxia?
Heart complications, such as cardiomyopathy and arrhythmias, are a primary concern for people living with FRDA. A cardiologist will perform regular EKGs and echocardiograms to monitor your heart health and ensure any issues are treated early.
What is the mFARS scale used for?
The modified Friedreich Ataxia Rating Scale (mFARS) is a standardized tool used by neurologists to track the progression of neurological symptoms over time. It helps doctors measure your physical changes and evaluate how well treatments are working.
What are the potential side effects of Skyclarys?
While generally well-tolerated, Skyclarys carries a risk of fluid retention, which can be dangerous for patients with underlying heart conditions. You should report any unexpected swelling, shortness of breath, or sudden weight gain to your doctor immediately.

Questions to Ask Your Doctor

Curated prompts to bring to your next appointment.

  1. 1.Are you familiar with the modified Friedreich Ataxia Rating Scale (mFARS) to track my progression?
  2. 2.Does our local center participate in the Collaborative Clinical Research Network (CCRN) for Friedreich Ataxia?
  3. 3.Is the patient currently a candidate for Skyclarys (omaveloxolone), and what baseline tests (like liver enzymes) are needed?
  4. 4.Can you refer me to a cardiologist and an orthopedist who have specifically treated patients with Friedreich Ataxia before?
  5. 5.What is our plan for a swallow study or speech evaluation to monitor for silent aspiration?

Questions For You

Tap a prompt to share your answer — we'll use it plus this page's context to start a tailored conversation.

References

References (21)
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    Friedreich Ataxia.

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    Poincaré plot analysis of electrocardiogram uncovers beneficial effects of omaveloxolone in a mouse model of Friedreich's ataxia.

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    Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension.

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    Characteristics of Adverse Events and Clinical Risks of Omaveloxolone Based on FAERS Data.

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    Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatment.

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This page provides educational information about Friedreich Ataxia treatments and care team structures. Always consult your neurologist or a specialized ataxia care center for medical advice tailored to your specific symptoms and condition.

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