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Approval of omaveloxolone for Friedreich ataxia

The recent approval of omaveloxolone for the treatment of Friedreich ataxia in the USA and Europe represents an important milestone in the field of rare neurological diseases. However, many challenges lie ahead, including the translation of trial results into clinical practice, and the management of patients’ expectations.

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Acknowledgements

S.B. and E.I. are members of the European Reference Network for Rare Neurological Diseases — project ID 739510.

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Correspondence to Sylvia Boesch.

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Competing interests

S.B. reports consultancy activity for VICO Therapeutics, REATA Pharmaceutics and Biogen, advisory board activity for REATA Pharmaceutics and Biogen, and honoraria from Ipsen, Merz Pharma, Abbvie, REATA Pharmaceutics and Biogen. E.I. declares no competing interests.

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Friedreich’s Ataxia Treatment Pipeline: https://www.curefa.org/research/research-pipeline

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Boesch, S., Indelicato, E. Approval of omaveloxolone for Friedreich ataxia. Nat Rev Neurol 20, 313–314 (2024). https://doi.org/10.1038/s41582-024-00957-9

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