Wednesday, June 18, 2025

Friedreich's ataxia—a rare multisystem disease

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Friedreich's ataxia—a rare multisystem disease, Reetz, KathrinCosta, Ana Sofia et al., The Lancet Neurology, Volume 24, Issue 7, 614 - 6...
Sunday, June 15, 2025

Sulforaphane Targets Multiple Pathological Processes in Friedreich Ataxia Patient-Induced Pluripotent Stem Cell-Derived Sensory Neurons

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Yang W, Thompson B, Miellet S, Maddock M, Napierala M, Dottori M, Kwa F. Sulforaphane Targets Multiple Pathological Processes in Friedreich ...
Friday, June 13, 2025

Omaveloxolone, But Not Dimethyl Fumarate, Improves Cardiac Function in Friedreich's Ataxia Mice With Severe Cardiomyopathy

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Omaveloxolone, But Not Dimethyl Fumarate, Improves Cardiac Function in Friedreich's Ataxia Mice With Severe Cardiomyopathy Lili Salinas ...
Thursday, June 12, 2025

Cerebellar transcranial direct current stimulation in Friedreich ataxia: anatomy matters

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Roderick P.P.W.M. Maas, Dennis J.L.G. Schutter, Cerebellar transcranial direct current stimulation in Friedreich ataxia: anatomy matters, Cl...
Thursday, June 5, 2025

Design Therapeutics Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2

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CARLSBAD, Calif., June 04, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. today announced that the first Friedreich ataxia (FA) patient ...
Tuesday, June 3, 2025

Managing Aminotransferase Elevations in Patients with Friedreich Ataxia Treated with Omaveloxolone: A Review and Expert Opinion on Use Considerations

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Authors: Susan Perlman, Mathieu Anheim, Sylvia Boesch, James H. Lewis, David R. Lynch. Published in: Neurology and Therapy. doi:10.1007/s401...

EMA approved orphan medicines since the implementation of the orphan legislation

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Hahl, E., Kurko, T., Koskinen, H. et al. EMA approved orphan medicines since the implementation of the orphan legislation. Orphanet J Rare D...
Wednesday, May 28, 2025

Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatment

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Gunther, K., Profeta, V., Keita, M. et al. Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatm...
Monday, May 26, 2025

Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice

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Matuszek, Z., Arbab, M., Kesavan, M. et al. Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia re...

Impaired DNA double-strand breaks repair in Friedreich’s Ataxia fibroblasts

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Rafka Challita, Dana Tohme, Charbel Feghaly, Hanin Bou Hadir, Walaa Chebli, Elie Estephan, Rana El-Hassan, Sima Hussayni, Wassim Abou Kheir,...