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 ...
Saturday, August 6, 2022

Lexeo to start phase I/II clinical trial for patients with FA cardiomyopathy.

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5 ago 2022
Thursday, July 28, 2022

Importance of an echocardiogram in the evaluation of ataxia

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Stokreef S, Lemos M, Quintas S.; BMJ Case Reports CP 2022;15:e248691. doi:10.1136/bcr-2021-248691 We present the case of a boy in his middle...
Tuesday, July 19, 2022

Unleashing the potential of AAV gene therapy

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Biopharma Dealmakers (Biopharm Deal). 18/7/2022. Voyager Therapeutics is unlocking the potential of adeno-associated virus gene therapy to t...