Children's Hospital of Philadelphia
Philadelphia, United States
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Shown for Austria
What do you need help with?
- Join a trialStudies open to new patients in your country, and abroad if you want.3 open in Austria · 18 worldwide
- Offer tissue or dataGive a sample or your health data to research.
- Get supportPatient groups, money and work, help for family.3 places to get help in Austria
- Access and costGetting a treatment that is not on offer here, and who pays.
- Questions for your doctorReady lists to print or copy before your next visit.
- Don't pay for thisOffers with no proof in people, and what to check first.
Where treatment stands for Friedreich ataxia
Four steps, from proven to unproven.
Approved in Austria
We do not have the list of medicines approved in Austria yet. Your doctor knows what is available here.
Being tested in people
3 open trials in Austria, 18 worldwide. See the trials
- 0 Phase 1
- 0 Phase 2
- 0 Phase 3
- 3 Other studies
Phase 1 – first tests in people, mainly for safety · Phase 2 – does it help? · Phase 3 – compared with the usual treatment.
Trials taking new patients now. Each trial counts once; one in two phases counts as the higher one.
- Nomlabofusp (Phase 3)
- Omaveloxolone (Phase 3)
- Vatiquinone (Phase 3)
- CTI-1601 (Phase 2)
Only in the lab or in animals
Of the 20 newest articles, 3 were in animals and 0 in the lab. See them
No proof it works
We have not checked claims for this disease yet. Check a post or a product
Latest research
The five newest articles. The label comes from the PubMed index: whether it was tested in people, in animals or only in the lab.
- Not labelled yetAxial Function Reflects Global Severity in Inherited Ataxias: The Upright Stability Score in Ambulatory Friedreich vs. Spinocerebellar Ataxia.Ann Clin Transl Neurol · 2026-11-04
- ReviewOculomotor Phenomenology in Cerebellar Ataxias: A Video-Illustrated Practical Diagnostic Approach.Mov Disord Clin Pract · 2026-11-04
- Not labelled yetProvider Perspectives on Friedreich Ataxia: A Cross-Sectional Survey to Assess Disease Familiarity and Decision-Making in the US.Degener Neurol Neuromuscul Dis · 2026-10-24
- Not labelled yetEfficacy and Safety of Omaveloxolone in Friedreich Ataxia: Real-World Data from an Advanced Disease Cohort.Mov Disord Clin Pract · 2026-10-29
- In peopleIntrinsic Activity Reductions and Cerebello-Cortical Diaschisis-Like Mechanism in Friedreich Ataxia: An fALFF Resting-State fMRI Study.Brain Behav · 2026-11-01
Not labelled yet: PubMed has not indexed the article yet, so we do not guess.
All articles further down this pageSupport near you
Patient groups and help in Austria.
- 1450 – Rund-um-die-Uhr-GesundheitsberatungAustria's nationwide 24/7 telephone health advice line: trained staff assess symptoms and guide callers to the appropriate care.
- Telefonseelsorge Österreich – Notruf 142Free, confidential 24/7 emotional support and crisis line for anyone in Austria, reachable on 142.
- Gesundheitsportal Österreich (gesundheit.gv.at)Austria's official public health portal, offering quality-assured information on diseases, prevention, health services and patient rights.
All supportOrganisations that can help, further down this page
Cure status
- NomlabofuspPhase 3 · NCT07778836
- OmaveloxolonePhase 3 · NCT06054893 · NCT06953583
- VatiquinonePhase 3 · NCT07681713 · NCT05515536
- CTI-1601Phase 2 · NCT06447025
- GV101Phase 2 · NCT07870590
- LX2006Phase 2 · NCT07721025
- DT-216P2Phase 1/2 · NCT06874010
- High Dose LX2006Phase 1/2 · NCT05445323
- Low dose LX2006Phase 1/2 · NCT05445323
- Mid Dose LX2006Phase 1/2 · NCT05445323
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXNPhase 1 · NCT05302271
- PrednisonePhase 1 · NCT05302271
- SGT-212Phase 1 · NCT07180355
Closest to patients
- Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)Phase 3 trial: VatiquinoneUniversity of South Florida · Tampa, United States + 8 more hospitalsPhase 3Results expected 2029
- A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's AtaxiaPhase 3 trial: NomlabofuspLarimar Therapeutics, Inc. (sponsor) · 5 hospitals in France and United StatesPhase 3Results expected 2029
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If you are looking for a relative
Here you will find questions you can ask, help with difficult talks, organisations for carers and your relative's rights.
Questions for your relative's doctor
How do I talk to them about this?
You do not need the right words. Ask what your relative wants to know, and let them set the pace. These pages can help.
Support for you as a carer
Looking after someone is work too. These organisations help carers, and the list further down this page shows helplines by country.
Power of attorney and patient rights, by country
A doctor can share information and a relative can act only with the patient's consent or a legal authority. It is easier to arrange this early.
- Hungary: patient rights representatives (Integrált Jogvédelmi Szolgálat)
- Germany: power of attorney and care directives (Federal Ministry of Justice)
- Austria: adult protection and representation (Ministry of Justice)
- United Kingdom: lasting power of attorney (GOV.UK)
- EU: your rights to healthcare in another EU country (Your Europe)
If you are looking for your child
Children are often treated differently from adults, in specialist centres. Here is where to start.
Before you pay
Parents often receive offers of paid treatment. Please go through this list before you pay for anything.
For patient organisations
You can share or print these pages for your members. They are free and need no account.
Apply for experimental treatment or a research study
The trial team decides who can take part; your doctor can help you ask.
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Latest news
Results, new trials and publications, newest first. Every item links to its source.
Results and new studies, newest first, in plain words.
Plain-language summary pending. Original title shown.
New and changed trials of the last 30 days, without an account and without tracking.
RSS feedCalendar reminderPublication
Axial Function Reflects Global Severity in Inherited Ataxias: The Upright Stability Score in Ambulatory Friedreich vs. Spinocerebellar Ataxia.
Axial Function Reflects Global Severity in Inherited Ataxias: The Upright Stability Score in Ambulatory Friedreich vs. Spinocerebellar Ataxia.
Annals of clinical and translational neurology
PubMed · PMID 42984128Publication
Intrinsic Activity Reductions and Cerebello-Cortical Diaschisis-Like Mechanism in Friedreich Ataxia: An fALFF Resting-State fMRI Study.
Intrinsic Activity Reductions and Cerebello-Cortical Diaschisis-Like Mechanism in Friedreich Ataxia: An fALFF Resting-State fMRI Study.
Brain and behavior
PubMed · PMID 42949167Publication
Provider Perspectives on Friedreich Ataxia: A Cross-Sectional Survey to Assess Disease Familiarity and Decision-Making in the US.
Provider Perspectives on Friedreich Ataxia: A Cross-Sectional Survey to Assess Disease Familiarity and Decision-Making in the US.
Degenerative neurological and neuromuscular disease
PubMed · PMID 42953761
Tools for your next step
Questions for your doctor
Pick up to 5 questions for your situation, then print them or take them on your phone.
Choose questionsOrganisations that can help
Helplines, patient groups and support services for you and your family.
Select your country to see the organisations there, starting with the ones closest to you.
Umbrella organisations (they connect national groups)
Euro-Ataxia
European federation of national ataxia associations (incl. Friedreich ataxia): shares information on care and research progress.
Visit site: Euro-Ataxia
EURORDIS – Rare Diseases Europe
Alliance of over 1,000 rare disease patient organisations, with a list of rare disease helplines by country.
Visit site: EURORDIS – Rare Diseases Europe
Friedreich's Ataxia Research Alliance
FA patient organisation (US-based, international research reach): FA information, newly-diagnosed guide, clinical trial finder, community events, research funding.
Visit site: Friedreich's Ataxia Research Alliance
General support
For anyone who is ill, whatever the disease: patients' rights and legal aid, crisis lines, hospice and palliative care, care in another EU country, and patient organisations' umbrella bodies.
International
European Patients' Forum (EPF)
Umbrella of European patient organisations and national patient coalitions.
Visit site: European Patients' Forum (EPF)
International Alliance of Patients' Organizations
Global alliance of patient organisations of all diseases, promoting patient-centred healthcare.
Visit site: International Alliance of Patients' Organizations
Frontlight is not affiliated with these organisations; we list them because they help.
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Find fellow researchers
Groups with active Friedreich ataxia work.
University of Pennsylvania
Philadelphia, United States
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RWTH Aachen University
Aachen, Germany
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Centre National de la Recherche Scientifique
Paris, France
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The University of Texas Southwestern Medical Center
Dallas, United States
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German Center for Neurodegenerative Diseases
Bonn, Germany
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The University of Melbourne
Melbourne, Australia
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Murdoch Children's Research Institute
Melbourne, Australia
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Inserm
Paris, France
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Monash University
Melbourne, Australia
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Sorbonne Université
Paris, France
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Massachusetts General Hospital
Boston, United States
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