Friedreich Ataxia Scientific News

Friedreich Ataxia and close related scientific news. Topics related to rare diseases.

Tuesday, January 30, 2024

Protective effect of FXN overexpression on ferroptosis in L-Glu-induced SH-SY5Y cells

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Mengran Wang, Tingting Xuan, Haining Li, Jing An, Tianhui Hao, Jiang Cheng, Protective effect of FXN overexpression on ferroptosis in L-Glu-...
Friday, January 26, 2024

Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression

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Anti-gene oligonucleotides targeting Friedreichs ataxia expanded GAA:TTC repeats increase Frataxin expression, Negin Mozafari, Salome Isabel...

Omaveloxolone for the treatment of Friedreich Ataxia: clinical trial results and practical considerations

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David R Lynch, Susan Perlman & Kim Schadt (2024) Omaveloxolone for the treatment of Friedreich Ataxia: clinical trial results and practi...

Emerging antioxidant therapies in Friedreich's ataxia

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Fred J. Edzeamey, Zenouska Ramchunder, Charareh Pourzand, Sara Anjomani Virmoun, Front. Pharmacol. Sec. Experimental Pharmacology and Drug D...
Thursday, January 25, 2024

Omaveloxolone: a groundbreaking milestone as the first FDA-approved drug for Friedreich ataxia

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Pilotto, Federica et al., Trends in Molecular Medicine, Volume 0, Issue 0. doi:10.1016/j.molmed.2023.12.002   Omaveloxolone represents the f...
Tuesday, January 23, 2024

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of friedreich’s ataxia

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Chang, J. C., Ryan, M. R., Stark, M. C., Liu, S., Purushothaman, P., Bolan, F., Johnson, C. A., Champe, M., Meng, H., Lawlor, M. W., Halawan...
Friday, January 19, 2024

METTL17 is an Fe-S cluster checkpoint for mitochondrial translation

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METTL17 is an Fe-S cluster checkpoint for mitochondrial translation. Tslil Ast,Yuzuru Itoh,Shayan Sadre,Jason G. McCoy,Gil Namkoong,Jordan C...
Wednesday, January 17, 2024

Gene editing improves Endoplasmic reticulum-mitochondrial contacts and Unfolded Protein Response in Friedreich's Ataxia iPSC-derived neurons

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Priyanka Mishra, Anusha Sivakumar, Avalon Johnson, Carla Pernaci, Anna S. Warden, Lilas Rony El- Hachem, Emily Hansen1, Rafael A. Badell-G...
Saturday, January 13, 2024

Iron imbalance in neurodegeneration

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Levi, S., Ripamonti, M., Moro, A.S. et al. Iron imbalance in neurodegeneration. Mol Psychiatry (2024). doi:10.1038/s41380-023-02399-z   This...
Friday, January 12, 2024

Evaluating the therapeutic efficacy of SKYCLARYS: a promising FDA-approved drug for Friedreich ataxia treatment

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 Ahmed, Usaid MBBSa; Afaq, Laiba MBBSa; Muhammad, Aqeel MBBSa; Riaz, Rumaisa MBBSa; Akilimali, Aymar MDb. Evaluating the therapeutic efficac...
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