Drug repurposing in hereditary ataxia

Hereditary ataxias are rare disorders, and a context where small cohorts and high development costs make drug repurposing a pragmatic route. Technological advances are creating opportunities to complement pathway-guided plausibility with more target-supported and AI-nominated mechanistically grounded candidates. Also, digital twin and The Virtual Brain frameworks should be viewed in hereditary ataxias as future-oriented research tools to integrate multimodal data, support stratification, and generate testable system-level predictions in rare cohorts.