Friedreich ataxia: who works on it

One card per institution. Pick a target or a country to narrow the list.

• All targets
• NRF2 0
• BRD4 0
• KIT 0
• NTRK 0
• PPAR 0
• RUNX1 0
• STING 0

24 of 84 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• European Commission (Horizon Europe) · HORIZON-ERC

  Engineering Synthetic Gene Circuits through Competitive Dimerization. (DIMERCIRCUITS)

  Fondazione Telethon Ets· Roma, Italy· EU contribution €2,484,115· until Dec 2030

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  Discrete topological and conformal quantum field theory through combinatorial factorisation algebras (DisQ FA)

  Aalto Korkeakoulusaatio Sr· Espoo, Finland· EU contribution €226,277· until Aug 2027· 2 countries

• European Commission (Horizon Europe) · HORIZON-ERC

  Genomic observation and perturbation: genetic modifiers to untangle disease mechanisms of RFC1 repeat expansion (GEMREX)

  University College London· London, United Kingdom· EU contribution €1,500,000· until Apr 2030

• European Commission (Horizon Europe) · HORIZON-RIA

  PHOENIX: Precision Human Organs-on-chip for Enhanced Neuro-muscular and Cardiac Studies

  Politecnico di Milano· Milano, Italy· EU contribution €7,095,163· until Dec 2028· 5 countries

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-ERC

  Illuminating human Fe-S cluster biology (FeSurveil)

  Weizmann Institute of Science· Rehovot, Israel· EU contribution €1,499,999· until Aug 2029

5 of 7 running projects, newest first. Listed amounts together: €18.2m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Terminated

  A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

  NCT06681766· Larimar Therapeutics, Inc.· Updated 10 Jul 2026

  “Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.”Registry text, quoted unchanged. Source

• Withdrawn

  A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

  NCT06483802· Astellas Gene Therapies· Updated 28 Nov 2025

  “Corporate decision to withdraw the trial at this time.”Registry text, quoted unchanged. Source

• Withdrawn

  Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

  NCT06560346· Friedreich's Ataxia Research Alliance· Updated 8 Jun 2025

  “Lack of Funding”Registry text, quoted unchanged. Source

• Withdrawn

  Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

  NCT03761511· RWTH Aachen University· Updated 24 Jan 2024

  “Due to a temporary halt the recruitment start was delayed. A re-evaluation of the sponsor revealed, that the study is no longer feasible due to increased administrative and financial requirements.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.

  NCT07095062· IRCCS Eugenio Medea· Source

• Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia

  NCT06692296· IRCCS Eugenio Medea· Source

• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

  NCT01793168· Sanford Health· Source

• A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World

  NCT07887828· Biogen· Source

• A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

  NCT06953583· Biogen· Source

• Cardiac Output and Fatigue in Friedreich's Ataxia

  NCT07444333· Scott Barbuto· Source

• A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

  NCT02255435· Biogen· Source

• A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

  NCT06054893· Biogen· Source

• A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

  NCT06628687· Biogen· Source

• A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

  NCT06623890· Biogen· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Organoids 2
• CRISPR-Cas Systems 3
• Gene Expression Profiling 1
• Whole Genome Sequencing 1
• Exome Sequencing 1
• Immunohistochemistry 1
• Magnetic Resonance Imaging 5
• Machine Learning 1
• Cohort Studies 1

• Unrecognized high prevalence of expanded composite repeats in Friedreich ataxia.

  Hum Mol Genet, Feb 2026

See which targets of this disease your abstract mentions, and go to the people working on them.