Sunday, November 5, 2017

Insights on the conformational dynamics of human frataxin through modifications of loop-1

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Martín E. Noguera, Martín Aran, Clara Smal, Diego S. Vazquez, María Georgina Herrera, Ernesto A. Roman, Nadine Alaimo, Mariana Gallo, Javier...
Saturday, November 4, 2017

Teaching Video NeuroImages: Spastic ataxia syndrome: The Friedreich-like phenotype of ARSACS

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Saffie P, Kauffman MA, Fernandez JM, Acosta I, Espay AJ, de la Cerda A.;  Neurology. 2017 Oct;89(14) e178-e179. doi:10.1212/wnl.000000000000...
Friday, November 3, 2017

BioWatch Thoughts on BioMarin's Q3-2017 Update

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BioPortfolio, 30 Oct 2017 | Biotech Watcher BMN-290 to Treat Friedreich’s Ataxia (FA) BioMarin announced that it is advancing BMN-290 in...
Thursday, November 2, 2017

Personality and Neuropsychological Profiles in Friedreich Ataxia

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Sabrina Sayah, Jean-Yves Rotgé, Hélène Francisque, Marcela Gargiulo, Virginie Czernecki, Damian Justo, Khadija Lahlou-Laforet, Valérie Hahn,...
Wednesday, November 1, 2017

First Gene Therapy Treatment Candidate to Receive Orphan Designation in EU and USA

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Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich Ataxia. CAMBRIDGE, Mass.--(...
Tuesday, October 31, 2017

Gene therapy—from small beginnings to where we are now

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J C Glorioso and N Lemoine; Gene Therapy (2017) 24, 495–496; doi:10.1038/gt.2017.23 While there have been many hurdles to overcome that re...
Monday, October 30, 2017

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich’s ataxia

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Celine J. Rocca, Spencer M. Goodman, Jennifer N. Dulin, Joseph H. Haquang, Ilya Gertsman, Jordan Blondelle, Janell L. M. Smith, Charles J. H...
Sunday, October 29, 2017

Can rehabilitation improve the health and well-being in Friedreich’s ataxia: a randomized controlled trial?

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Milne SC, Corben LA, Roberts M, Murphy A, Tai G, Georgiou-Karistianis N, Yiu EM, Delatycki MB; Clin Rehabil. 2017 Oct 1:269215517736903. doi...
Wednesday, October 25, 2017

Reata Announces First Patient Enrolled in Part 2 of MOXIe Study of Omaveloxolone for the Treatment of Friedreich’s Ataxia

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IRVING, Texas, Oct. 23, 2017 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (NASDAQ:RETA) (“Reata” or the “Company”) today announced the en...
Tuesday, October 24, 2017

Friedreich’s ataxia: clinical features, pathogenesis and management

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A Cook, P Giunti; British Medical Bulletin,  2017, 1–12 doi:10.1093/bmb/ldx034 The last decade has seen important advances in our understa...