Top 5 Friedreich’s ataxia news stories of 2025
Double dose of Skyclarys news, gene therapy trials among highlights
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The team at Friedreich’s Ataxia News spent 2025 bringing readers the latest updates on research, treatments, and clinical trials related to the rare inherited disease. From long-term real-world outcomes with approved therapies to promising steps toward gene therapy, this year delivered meaningful progress for the Friedreich’s ataxia (FA) community.
Here, we’re taking a look back at the five most-read articles of the year, highlighting the stories that resonated most with our readers.
No. 5 – FDA clears Phase 1 trial for FA gene therapy SGT-212
Solid Biosciences has received clearance from the U.S. Food and Drug Administration (FDA) to begin clinical testing of its experimental gene therapy SGT-212 for FA. The therapy is designed to deliver a functional copy of the FXN gene, enabling cells to produce a protein whose deficiency in people with FA drives disease symptoms. SGT-212 employs a dual route of administration to target both the heart and specific brain regions commonly affected in FA. The Phase 1b trial, known as FALCON, is now underway and is expected to enroll about 10 FA patients, ages 18 to 40. The study’s primary goal is to assess safety in the first year after the gene therapy is administered.
No. 4 – Skyclarys slows FA progression over four years
Daily treatment with the approved oral therapy Skyclarys (omaveloxolone), marketed by Biogen, continued to slow disease progression in people with FA over four years, according to a presentation at the 2025 International Congress of Parkinson’s Disease and Movement Disorders in Honolulu, Hawaii. The findings are based on an extension of a Phase 2 trial that included participants who either continued Skyclarys after completing the original study or initiated the treatment during the extension. Compared with FA progression in natural history studies, patients receiving Skyclarys showed a lesser decline in neurological function, supporting the treatment’s long-term effectiveness. Another presentation at the event showed that investigational oral therapy vatiquinone, developed by PTC Therapeutics, led to significantly slower disease progression in adults with FA relative to untreated natural history, according to results from a separate Phase 2 trial.
No. 3 – Skyclarys tied to recovery of sensory nerve activity in sisters with FA
Treatment with Skyclarys improved peripheral sensory nerve function in two young sisters diagnosed with FA. Peripheral sensory nerves are located outside the brain and spinal cord, carrying bodily sensations, such as touch and pain, to the brain. Before treatment, sensory nerve signals were absent in both sisters, but they became detectable after six months on Skyclarys, suggesting the therapy may have a favorable impact on peripheral nerves in people with FA.
No. 2 – Voyager’s FA gene therapy poised for clinical testing in 2026
An experimental gene therapy for FA is expected to move into clinical testing this year. The therapy candidate, being developed by Voyager Therapeutics in collaboration with Neurocrine Biosciences, is designed to deliver a healthy copy of the FXN gene to the body’s cells, enabling production of a functional frataxin protein. Because the therapy can enter the brain, it may have the potential to address neurological complications of FA. Voyager said it planned to submit a regulatory application to the FDA seeking permission to begin testing the therapy in people.
No. 1 – Common antioxidant shows potential to increase frataxin in FA
Supplementation with the naturally occurring antioxidant alpha-lipoic acid (ALA) may boost production of frataxin. In the lab, treatment with ALA improved cellular function and reduced signs of oxidative stress, a type of cellular damage, both of which are typical features of FA. While the findings are limited to laboratory models, further studies are needed to assess the potential clinical benefits. The results, however, point to ALA’s potential as a therapeutic approach for FA, the researchers said.
We hope that these stories, along with all our reporting at Friedreich’s Ataxia News, have provided helpful information to the FA community throughout 2025. We look forward to continuing to serve as a trusted source of news and insights in the year ahead, and we wish all our readers a happy and healthy 2026.
