357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history

357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history. Vagabov, A. et al. Neuromuscular Disorders, Volume 53, 106087 DOI:10.1016/j.nmd.2025.106087 

 The results of the extension studies provide further evidence of the potential benefit of vatiquinone for the treatment of FA. The pre-specified endpoints for two different long-term extension studies were met, with highly statistically significant evidence of durable treatment benefit in slowing disease progression in paediatric and adult patients.

357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history