A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),Neurology
Link
https://link.springer.com/content/pdf/10.1007/s12311-023-01568-8.pdf
Reference16 articles.
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2. Babcock M, de Silva D, Oaks R, Davis-Kaplan S, Jiralerspong S, Montermini L, Pandolfo M, JKaplan J. Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin. Science. 1997;276:1709–12.
3. Delatycki MB, Bidichandani SI. Friedreich ataxia - pathogenesis and implications for therapies. Neurobiol Dis. 2019;132:104606.
4. Rummey C, Corben LA, Delatycki M, Wilmot G, Subramony SH, Corti M, Bushara K, Duquette A, Gomez C, Hoyle JC, et al. Natural history of Friedreich’s ataxia: heterogeneity of neurological progression and consequences for clinical trial design. Neurology. 2022;99:e1499–510.
5. Reetz K, Dogan I, Hilgers R-D, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesch S, et al. Progression characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study. Lancet Neurol. 2021;20:362–72.
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