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...
Wednesday, December 27, 2023

Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

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Arthur Lenahan, Sho Yano, Brett Graham, Kuntal Sen, Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA...
Tuesday, December 26, 2023

CO105 A Retrospective Study Characterizing Age at Loss of Ambulation Among Patients With Friedreich Ataxia Using Health Administrative Claims Data in the United States

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A. Salvucci, C. Qian, L. Powell, D. Lynch, G. Vasco, K. Johnston, I. Tomazos, CO105 A Retrospective Study Characterizing Age at Loss of Ambu...

Mitochondrial impairment, decreased sirtuin activity and protein acetylation in dorsal root ganglia in Friedreich Ataxia models

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Arabela Sanz-Alcázar, Elena Britti, Fabien Delaspre, Marta Medina-Carbonero, Maria Pazos-Gil, Jordi Tamarit, Joaquim Ros & Elisa Cabisco...

Predictors of Survival in Friedreich's Ataxia: A Prospective Cohort Study

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Indelicato, E., Reetz, K., Maier, S., Nachbauer, W., Amprosi, M., Giunti, P., Mariotti, C., Durr, A., de Rivera Garrido, F.J.R., Klopstock, ...