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...
Thursday, January 11, 2024

Tissue Iron in Friedreich Ataxia

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Arnulf H Koeppen. Tissue Iron in Friedreich Ataxia. J. Integr. Neurosci. 2024, 23(1), 4. Doi:10.31083/j.jin2301004 (registering DOI)   Iron ...
Tuesday, January 9, 2024

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

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SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc.  FDA meeting for vatiquinone to discuss how the MOVE-FA data alo...
Saturday, January 6, 2024

Perspectives of the Friedreich ataxia community on gene therapy clinical trials

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Shandra J. Trantham,Mackenzi A. Coker,Samantha Norman,Emma Crowley,Julie Berthy,Barry J. Byrne,Sub Subramony,XiangYang Lou,Manuela Corti; Mo...
Wednesday, January 3, 2024

Omaveloxolone ameliorates isoproterenol-induced pathological cardiac hypertrophy in mice

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Xianchao Li, Yang Wu, Yunzhao Yang, Yaohua Wu, Xi Yu & Wenjuan Hu (2023) Omaveloxolone ameliorates isoproterenol-induced pathological ca...