Friedreich ataxia
An inherited condition that gets worse over time and affects balance and coordination; signs can include weak legs and slurred speech. There is usually no cure for ataxia, so treatment eases the symptoms with therapy and medicines. It usually starts before age 25, and many people need a wheelchair 10 to 20 years after it begins. Many people also get heart problems, and some get diabetes or a curved spine.
Read more about Friedreich ataxiaTrial-stage treatments and research studies you can join
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Where treatment stands
There is no cure in most cases, but treatment can control symptoms, and a first medicine for people aged 16 and over has been approved in the UK.
Approved: 1
Officially approved for this disease in the EU, the US, Switzerland or France.
Human testing stage: 2
Being tested in people. Many treatments at this stage are never approved.
Animal testing stage: 5
Worked in animals in a study published in the last 12 months. Most treatments at this stage never reach people.
Placebo: a dummy treatment without the medicine, used for comparison.
Phase 1, 2, 3: the steps of testing in people. Phase 1 checks safety, phase 3 is the last step before approval.
The EU medicines agency (EMA) and its list of medicines in the EU, US prescribing labels (DailyMed, openFDA), the Swiss medicines register (Swissmedic), the French medicines database (ANSM) and the Health Canada drug database, checked on 11 November 2026. Every medicine links to its own official text.
A computer made this list from the official texts: it kept only medicines whose own approval text names the disease, wrote "Who it is for" from that text and checked it against the text a second time. People have not reviewed it yet.
The trials: ClinicalTrials.gov and the EU trials register, as copied into our trial database, checked on 12 November 2026. Every treatment in trials links to its own trial records; the texts are written by AI from them and checked against them by a second AI. People have not reviewed them yet. Placebo, standard medicines used for comparison and procedures are left out.
Phase 3 trials are listed from every country; only some phase 2 trials are listed, so 3 other open phase 2 trials are not listed.
Animal and lab research: studies listed in PubMed and published in the 12 months before 12 November 2026, found by a search for Friedreich's ataxia. AI chose the treatments from the study summaries and wrote the texts; a second AI checked each one against its study summary. People have not reviewed them yet. Treatments already in the approved or human testing lists are left out. A result in animals or cells does not show that a treatment works in people.
Not medical advice. Talk to your doctor before you change any treatment.
The approval records under a medicine come from Drugs@FDA and the FDA labels (openFDA) and from the EU medicines agency’s medicine data, with its orphan designations, checked on 13 Nov 2026. A date is the medicine’s first approval, for any use; the quoted text is its approved use as written today.
Support organisations
Patient organisations and helplines in Poland, for you and your family.
We have no organisations for this disease listed in Poland yet. General support there is below.
General support in Poland
- Centrum Wsparcia dla osób dorosłych w kryzysie psychicznym
Free 24/7 support line (800 70 2222) and chat for adults in psychological crisis in Poland, funded by the Ministry of Health.
- pacjent.gov.pl
Patient portal of the Polish Ministry of Health and National Health Fund with reliable, verified information on health and the care system.
- Rzecznik Praw Pacjenta
Poland's central government Patient Ombudsman protecting patient rights, with a free patient helpline (800 190 590).
International organisations
- National Ataxia Foundation
US nonprofit empowering people affected by ataxia, including Friedreich's ataxia, with education, support groups and research.
Offer tissue or data
There are three ways to help research move faster.
Approved treatments
1 medicine is approved for Friedreich ataxia in at least one of the places we checked. An approval in one place does not mean the medicine is offered or paid for where you live.
Checked on 11 Nov 2026 in the official lists of the US (FDA), the EU (EMA), Japan (PMDA, approvals since April 2004), Switzerland (Swissmedic), France (ANSM, medicines approved in France only) and Taiwan (TFDA). For Canada (Health Canada) and Singapore (HSA) we checked only whether a medicine is sold there, not what for. Other countries' own approvals were not checked.
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“Cures” others already fact-checked
- Omega-3 fatty acids (fish oil) & Friedreich ataxia
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About Friedreich ataxia
Sources: NHS, MedlinePlus Genetics, MHRA
An inherited condition that gets worse over time and affects balance and coordination; signs can include weak legs and slurred speech. There is usually no cure for ataxia, so treatment eases the symptoms with therapy and medicines. It usually starts before age 25, and many people need a wheelchair 10 to 20 years after it begins. Many people also get heart problems, and some get diabetes or a curved spine.
What is it?
Friedreich ataxia is caused by a change in a gene called FXN. The gene gives instructions for making frataxin, a protein that is important for mitochondria, the energy-producing centers within cells. In people with the condition, a small part of the gene is repeated far too many times, so the body makes too little frataxin. Certain nerve and muscle cells cannot work properly when there is too little frataxin.
It is the most common inherited ataxia, but still rare: at least 1 in 50,000 people, and about 1 in 40,000 in the US. Most people begin to have symptoms between ages 5 and 15; about 1 in 4 people have a form in which symptoms begin after age 25. A child gets it only when both parents pass on the altered gene; the parents are usually healthy carriers.
The first signs
Poor coordination and balance are often the first signs you notice.
- unsteadiness, clumsiness and falling often;
- speech that becomes slow and slurred;
- weak legs;
- less feeling in the hands and feet.
How doctors find out
Your family doctor asks about your symptoms, any ataxia in the family, alcohol and medicines, and checks your balance, walking and coordination. You are referred to a neurologist, or a paediatrician for children, for further tests; a genetic blood test can find the gene change responsible for Friedreich's ataxia. Other tests may include an MRI scan of the brain, nerve and muscle tests, and heart tests (ECG and an ultrasound scan of the heart).
Current treatment
A care team makes a care plan with you: physiotherapy, speech and occupational therapy, and medicines for muscle stiffness, bladder problems and nerve pain; if you develop heart muscle damage (cardiomyopathy), you have regular heart check-ups.
Will it get worse?
Yes, the symptoms usually get gradually worse over many years. Signs can also include difficulty swallowing, curving of the spine (scoliosis), and total or partial vision loss and hearing loss. Some people get diabetes. Many get a thickened heart muscle, which can cause chest pain, breathlessness and an uneven heartbeat, and can be life-threatening. Many people need a wheelchair 10 to 20 years after it starts. People with the condition tend to have a shorter life expectancy than normal; many people live until at least their 30s, and some can live into their 60s or beyond.
Medicines: what helps, and what to watch for
Omaveloxolone (Skyclarys), a capsule, is the first medicine approved for Friedreich ataxia, for people aged 16 and over. In a trial of 103 people aged 16 to 40, it reduced physical problems more than a dummy medicine after 48 weeks. Other medicines ease single symptoms: muscle relaxants such as baclofen or tizanidine, or botulinum toxin injections, for stiff muscles; medicines for bladder problems; and amitriptyline, gabapentin or pregabalin for nerve pain.
Good to know:
- damage to the heart muscle (cardiomyopathy) is a common problem in some types of ataxia; if you develop it, you will have regular check-ups with a heart specialist and may need medicine;
- ordinary painkillers such as paracetamol or ibuprofen are not usually effective for nerve pain;
- for bladder problems, limiting how much you drink during the day, planning regular trips to the toilet and avoiding drinks such as caffeine and alcohol can help in some cases;
Exercise and therapy help too
A team, often a neurologist, a physiotherapist and a specialist nurse, makes a care plan with you.
- daily physiotherapy exercises keep your muscles strong and supple; walking aids can help;
- a speech and language therapist can help with slurred speech and swallowing problems; your diet may need to include food that is easier to swallow;
- an occupational therapist helps with daily tasks, wheelchair use and changes at home, such as rails or a stairlift;
- a physiotherapist may be able to help you build up your stamina, and an occupational therapist can advise you how to adapt your daily activities, to help you cope with tiredness.
Is there a cure?
No, in most cases there is no cure for ataxia. In most cases there is no cure, and supportive treatment to control the symptoms is needed. Omaveloxolone is the first medicine approved for the condition itself, for people aged 16 and over.
Median survival
No reliable published figure was found.
Sources
Written in plain words by Frontlight from these pages: