Saturday, September 6, 2025

Characterizing Population Pharmacokinetics of Vatiquinone in Healthy Volunteers and Patients with Friedreich’s Ataxia

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Hu, Y.; Gao, L.; Lee, L.; Cherry, J.J.; Kong, R. Characterizing Population Pharmacokinetics of Vatiquinone in Healthy Volunteers and Patient...
Thursday, September 4, 2025

Repeat-associated ataxias in a German patient cohort analysed by targeted parallel long-read sequencing

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Hannes Erdmann, Annalisa Schaub, Morghan C Lucas, Veronika Scholz, Anna Benet-Pages, Kerstin Becker, Christine Dineiger, Veronika Mayer, Ing...
Tuesday, September 2, 2025

Design Therapeutics Faces Regulatory Hurdle While Maintaining Financial Stability

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01.09.25 09:19, Börse Global (en).  Clinical Program Pivot Following FDA Decision A significant development emerged from regulatory discuss...
Monday, September 1, 2025

Frataxin deficiency drives cardiac dysfunction and transcriptional dysregulation in Friedreich ataxia iPSC model

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Frataxin deficiency drives cardiac dysfunction and transcriptional dysregulation in Friedreich ataxia iPSC model. Jarmon G. Lees, Haoxiang Z...
Sunday, August 31, 2025

In vivo gene therapy: A strategy for mutations, degenerations, and tumors

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Tao Wang, Mingyang Yu, Ping Liu, Zhiqiang Song, Cheng Li, Jianmin Yang, Na Liu, In vivo gene therapy: A strategy for mutations, degeneration...

Spatial perspective taking is impaired in spinocerebellar ataxias and Friedreich ataxia

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Karamazovova, S., Laczó, M., Matuskova, V. et al. Spatial perspective taking is impaired in spinocerebellar ataxias and Friedreich ataxia. S...

Alpha–lipoic acid supplementation improves pathological alterations in cellular models of Friedreich ataxia

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Talaverón-Rey, M., Reche-López, D., Povea-Cabello, S. et al. Alpha–lipoic acid supplementation improves pathological alterations in cellular...
Tuesday, August 19, 2025

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

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WARREN, N.J., Aug. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) has iss...
Monday, August 18, 2025

Propensity-matched analysis comparing omaveloxolone treatment to Friedreich ataxia natural history data: a plain language summary

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Lynch, D. R., Goldsberry, A., Rummey, C., Farmer, J., Boesch, S., Delatycki, M. B., … Meyer, C. (2025). Propensity-matched analysis comparin...

Sensory nerve action potential reappearance after omaveloxolone treatment in patients with Friedreich ataxia

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Sensory nerve action potential reappearance after omaveloxolone treatment in patients with Friedreich ataxia. Melita Rotar, Lea Leonardis; C...