Wednesday, March 22, 2023

Assessment of Hepatic Safety in Patients with Friedreich’s Ataxia in the MOXIe Trial of Omaveloxolone

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S.H. Subramony, MD, University of Florida Health System, David Lynch, MD, PhD, Children's Hospital of Philadelphia, Martin Delatycki, MD...

Assessment of Cardiac Safety in Patients with Friedreich’s Ataxia in the MOXIe Trial of Omaveloxolone

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S.H. Subramony, MD, University of Florida Health System, David Lynch, MD, PhD, Children's Hospital of Philadelphia, Martin Delatycki, MD...
Monday, March 20, 2023

Ataxia Scales for the Clinical Evaluation

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Bürk, K. (2023). Ataxia Scales for the Clinical Evaluation. In: Gruol, D.L., Koibuchi, N., Manto, M., Molinari, M., Schmahmann, J.D., Shen, ...
Saturday, March 18, 2023

A Novel Metric for Predicting Severity of Disease Features in Friedreich's Ataxia

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Rodden, L. N., Rummey, C., Kessler, S., Wilson, R. B., & Lynch, D. R. (2023). A Novel Metric for Predicting Severity of Disease Features...
Wednesday, March 15, 2023

Vesigen Therapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance (FARA) to Develop a Targeted Genome Editing Therapeutic Strategy

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March 14 2023. Vesigen Therapeutics, a biotechnology company developing targeted therapies by engineering a distinct class of human extracel...
Monday, March 6, 2023

Sensoren in der Kleidung plus KI: Erfolgversprechende Diagnosetechnologie: Bei Friedreich-Ataxie und Duchenne-Muskeldystrophie getestet

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Patzer, K.-H. (2023). Sensoren in der Kleidung plus KI: Erfolgversprechende Diagnosetechnologie: Bei Friedreich-Ataxie und Duchenne-Muskeldy...

CRISPR/Cas9-based edition of frataxin gene in Dictyostelium discoideum for Friedreich’s Ataxia disease modeling

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Gentili, H. G., Pignataro, M. F., Olmos, J., Pavan, M. F., Ibanez, L. I., Santos, J., & Velazquez, F. (2023). CRISPR/Cas9-based edition ...

Reduced cerebello-cerebral functional connectivity correlates with disease severity and impaired white matter integrity in Friedreich ataxia

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Kerestes, R., Cummins, H., Georgiou-Karistianis, N., Selvadurai, L. P., Corben, L. A., Delatycki, M. B., Egan, G. F., & Harding, I. H. (...
Saturday, March 4, 2023

Proprioceptors-enriched neuronal cultures from induced pluripotent stem cells from Friedreich ataxia patients show altered transcriptomic and proteomic profiles, abnormal neurite extension, and impaired electrophysiological properties

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Dionisi, C., Chazalon, M., Rai, M., Keime, C., Imbault, V., Communi, D., Puccio, H., Schiffmann, S. N., & Pandolfo, M. (2023). Proprioce...
Wednesday, March 1, 2023

FDA has approved Skyclarys (omaveloxolone) as the first treatment for Friedreich’s ataxia

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  FDA approves first treatment for Friedreich’s ataxia FDA Widens Path for Rare-Disease Treatments With New Approval Reata Pharmaceuticals A...