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...

Best Oculomotor Endpoints for Clinical Trials in Hereditary Ataxias: A Systematic Review and Consensus by the Ataxia Global Initiative Working Group on Digital‑Motor Biomarkers

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Pretegiani E, Garces P, Antoniades CA, Sobanska A, Kovacs N, Ying SH, Gupta AS, Perlman S, Szmulewicz DJ, Pane C, Németh AH, Jardim LB, Coar...
Sunday, August 17, 2025

FRIEDREICH ATAXIA- STEROIDOGENESIS (FRIEDSTERO)

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ClinicalTrials.gov ID NCT07123142. Sponsor Istanbul University Information provided by Ozge Bayrak Demirel, Istanbul University (Responsible...
Saturday, August 16, 2025

Larimar Therapeutics Reports Second Quarter 2025 Financial Results

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BALA CYNWYD, Pa., Aug. 14, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar). Initial data from the 50 mg dose in the open label...

Lexeo Therapeutics Reports Second Quarter 2025 Financial Results and Operational Highlights

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NEW YORK, Aug. 14, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc.​. Breakthrough Therapy designation granted for LX2006 based on interim ...