Ataxia Therapeutics: A Comprehensive Overview of the Clinical Development Pipeline. (PubMed, Pharmaceut Med) - Oct 25, 2026 - "Despite significant advances in molecular diagnostics and disease characterization, effective disease-targeted treatments remain limited, with omaveloxolone currently representing the only approved therapy for Friedreich's ataxia...More than half of investigational agents are repurposed compounds, reflecting a pragmatic strategy to accelerate therapeutic progress in rare disorders. Overall, the ataxia therapeutic landscape is expanding but remains fragmented, underscoring the need for improved patient stratification, validated biomarkers, harmonized registries, and international collaboration to enable the successful translation of emerging therapies into clinical practice."
Journal • Review • Ataxia • CNS Disorders • Friedreich ataxia • Gene Therapies • Movement Disorders • Rare Diseases
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Agata Polizzi; Elena Carbone; Eleonora Lacorte; Federica Perrone; Guido Bellomo; Marco Salvatore; Marisa Cappella; Nicola Vanacore; Nicoletta Locuratolo; Paola Piscopo
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