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...
Monday, October 23, 2017

Incidence et caractéristiques de la scoliose neurologique dans l’ataxie de Friedreich à maturité osseuse

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Jean Meyblum, Anne-Laure Simon, Christophe Vidal, Isabelle Husson, Bastien Roche, Brice Ilharreborde, Revue de Chirurgie Orthopédique et Tra...
Saturday, October 21, 2017

Cardiomyopathy in Friedreich’s Ataxia

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Pablo Salazar, Raksha Indorkar, Michael Dietrich, Afshin Farzaneh-Far; European Heart Journal, , ehx607, doi:10.1093/eurheartj/ehx607 A 27...
Friday, October 20, 2017

Nrf2-Inducers Counteract Neurodegeneration in Frataxin-Silenced Motor Neurons: Disclosing New Therapeutic Targets for Friedreich’s Ataxia

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Petrillo, S.; Piermarini, E.; Pastore, A.; Vasco, G.; Schirinzi, T.; Carrozzo, R.; Bertini, E.; Piemonte, F.; Int. J. Mol. Sci. 2017, 18, 2...
Thursday, October 19, 2017

Sustained FXN expression in dorsal root ganglia from a nonreplicative genomic HSV-1 vector

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Maria Ventosa, Zetang Wu and Filip Lim; The Journal of Gene Medicine, Accepted manuscript online: 17 OCT 2017 08:40PM EST DOI: 10.1002/jgm.2...
Wednesday, October 18, 2017

BioMarin Selects BMN 290 for Friedreich's Ataxia

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SAN RAFAEL, Calif., Oct. 18, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) updated the investment community on the Compan...