Friedreich Ataxia
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-08-19EMA Authorised: Skyclarys (omaveloxolone) — Biogen Netherlands B.V.
Skyclarys (omaveloxolone) has been authorised in the EU for Friedreich ataxia.
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EMA approval · 2026-08-04EMA Authorised: Skyclarys (omaveloxolone) — Biogen Netherlands B.V.
Authorisation of Skyclarys (omaveloxolone) for Friedreich ataxia by the EMA.
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Clinical Course Of Disease In Participants With FA-CM
The clinical trial NCT06865482 by Lexeo Therapeutics is currently recruiting to study the clinical course of disease in Friedreich ataxia wi…
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CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia [Phase 2, Phase 3]
A Phase 2/3 trial evaluating vatiquinone for Friedreich Ataxia has completed and posted results. The update confirms results availability as…
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FDA label_change · 2026-04-07FDA Approved: SKYCLARYS — Supplemental Application (Biogen)
The FDA approved Biogen's supplemental application for Skyclarys (omaveloxolone). This approval updates the labeling for this Friedreich's a…
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CLINICAL_TRIALS research · 2026-04-02Clinical Trial Update: Friedreich Ataxia Nerve Ultrasund
A clinical trial evaluating nerve ultrasound as a biomarker in Friedreich ataxia is currently recruiting. Sponsored by Centre Hospitalier Un…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
This clinical trial update outlines an ongoing study by DZNE evaluating patient-reported outcomes, health economic considerations, and psych…
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CLINICAL_TRIALS research · 2026-03-31Clinical Trial Update: Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia [Phase 1]
This clinical trial update reports a Phase 1a/1b study of AAVrh.10hFXN gene therapy for Friedreich's ataxia-related cardiomyopathy, currentl…
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: FRIEDREICH ATAXIA- STEROIDOGENESIS
A clinical trial investigating Friedreich's Ataxia and steroidogenesis conducted by Istanbul University has completed.
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia [Phase 2]
An open-label Phase 2 clinical trial evaluating CTI-1601 in Friedreich's ataxia is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE) [Phase 3]
The BRAVE Phase 3 trial is recruiting to evaluate omaveloxolone (BIIB141) for Friedreich's ataxia in children aged 2 to 15 and assess long-t…
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EMA approval · 2026-03-13EMA Authorised: Skyclarys (omaveloxolone) — Biogen Netherlands B.V.
The European Medicines Agency has authorised Skyclarys (omaveloxolone) for Friedreich ataxia, with Biogen Netherlands B.V. as the marketing …
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CLINICAL_TRIALS trial_result · 2026-03-11Clinical Trial Update: NAD+ and Exercise in FA [NA]
A Friedreich's ataxia trial evaluating NAD+ and exercise has completed and posted results.
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CLINICAL_TRIALS research · 2026-03-09Clinical Trial Update: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia [Phase 1]
A Phase 1 study of SGT-212 gene therapy for Friedreich's ataxia is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: Cardiac Output and Fatigue in Friedreich's Ataxia [NA]
Clinical trial update examining cardiac output and fatigue in Friedreich's Ataxia. The study is currently NOT_YET_RECRUITING.
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CLINICAL_TRIALS withdrawal · 2026-01-28Clinical Trial Update: A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia [Phase 1]
The Phase 1 trial of nomlabofusp in adolescents and children with Friedreich's Ataxia has been terminated.
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CLINICAL_TRIALS trial_result · 2026-01-16Clinical Trial Update: Measurement of Frataxin mRNA in Biofluids
This completed clinical trial from the University of Florida assesses measurement of frataxin mRNA levels in biofluids in individuals with F…
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CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: 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 [Phase 1]
Phase 1 study to assess how BIIB141 (Omaveloxolone) is processed in the body and its safety in children ages 2 to 15 with Friedreich's Ataxi…
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CLINICAL_TRIALS trial_result · 2025-12-23Clinical Trial Update: Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia [Phase 1, Phase 2]
A Phase 1/Phase 2 clinical trial of a gene therapy for cardiomyopathy associated with Friedreich's ataxia is currently active but not recrui…
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CLINICAL_TRIALS trial_result · 2025-12-12Clinical Trial Update: FRDA Investigator Initiated Study (IIS) With Elamipretide [Phase 1, Phase 2]
A Phase 1/Phase 2 investigator-initiated study of Elamipretide for Friedreich Ataxia has completed, with results posted on 2025-12-12.
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CLINICAL_TRIALS withdrawal · 2025-11-28Clinical Trial Update: A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia [Phase 1]
A Phase 1 trial of ASP2016 in adults with Friedreich Ataxia-associated cardiomyopathy has been withdrawn.
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CLINICAL_TRIALS trial_result · 2025-11-03Clinical Trial Update: Respiratory Training in Friedreich's Ataxia [NA]
A clinical trial evaluating respiratory training for Friedreich's Ataxia has completed.
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CLINICAL_TRIALS trial_result · 2025-10-30Clinical Trial Update: A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia [Phase 3]
Phase 3 study of Vatiquinone in Friedreich Ataxia is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-10-03Clinical Trial Update: Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
Update on the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) patient registry, noting that the study status is C…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: Biomarkers in Friedreich's Ataxia
A University of Florida-sponsored clinical trial is currently recruiting to study biomarkers in Friedreich's Ataxia.