Saturday, August 27, 2022

Preservation of bioenergetics and inhibition of ferroptosis with the novel compound SBT-588 in Friedreich’s ataxia cell models

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Laura E. Kropp, Hatim Zariwala, Yunmi Park, Martin Redmon, David A. Brown, Alyssa Handler; Biochimica et Biophysica Acta (BBA) - Bioenergeti...
Tuesday, August 23, 2022

Frataxin deficiency disrupts mitochondrial respiration and pulmonary endothelial cell function

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Miranda K Culley, Monica Mehta, Jingsi Zhao, Dror Perk, Yi Yin Tai, Ying Tang, Sruti Shiva, Marlene Rabinovitch, Mingxia Gu, Thomas Bertero,...
Saturday, August 20, 2022

A systematic review of disease prevalence, health-related quality of life, and economic outcomes associated with Friedreich’s Ataxia

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Katherine Buesch & Rongrong Zhang (2022) , Current Medical Research and Opinion, DOI: 10.1080/03007995.2022.2112870  Findings from this ...
Wednesday, August 17, 2022

Direct utility of natural history data in analysis of clinical trials: Propensity match-based analysis of Omaveloxolone in Friedreich ataxia using the FA-COMS dataset

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David Lynch, Angie Goldsberry, Christian Rummey, Jennifer Farmer, Sylvia Boesch, martin delatycki, Paola Giunti, j chad Hoyle, Caterina Mari...
Thursday, August 11, 2022

Replication dependent and independent mechanisms of GAA repeat instability

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Chiara Masnovo, Ayesha F. Lobo, Sergei M. Mirkin; DNA Repair, Volume 118, 2022, 103385, doi:10.1016/j.dnarep.2022.103385.  Here, we provide ...
Wednesday, August 10, 2022

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

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Kevin C. Kemp, Anastasia Georgievskaya, Kelly Hares, Juliana Redondo, Steven Bailey, Claire M. Rice, Neil J. Scolding, Chris Metcalfe & ...

FDA Extends Review of Omaveloxolone in Friedreich Ataxia

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August 9, 2022. After Reata Pharmaceuticals submitted an analysis from the MOXIe extension study of the investigational agent as part of the...
Tuesday, August 9, 2022

Reata continues to hit FDA roadblocks with its Friedreich’s ataxia candidate, noting regulator 'concerns'

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Endpoints News. August 8, 2022.  Reata Pharmaceuticals is facing an FDA wall on its small molecule candidate to treat Friedreich’s ataxia, o...

Design Therapeutics Highlights Upcoming Milestones and Reports Second Quarter 2022

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CARLSBAD, Calif., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company develop...
Sunday, August 7, 2022

Ataxia de friedreich, lucha por la supervivencia

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Beatriz Miguel Palacios. Diplomada en Fisioterapia. Hospital Universitario Miguel Servet. Zaragoza, Nuria Bedoya Chocán. Celadora. Hospital ...