Friday, November 17, 2017

Voyager Therapeutics "update"

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CAMBRIDGE, Mass., Nov. 16, 2017 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company focused ...
Wednesday, November 15, 2017

Proactive Ethical Design for Neuroengineering, Assistive and Rehabilitation Technologies: the Cybathlon Lesson

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Marcello Ienca, Reto W. Kressig, Fabrice Jotterand and Bernice Elger; ournal of NeuroEngineering and Rehabilitation 201714:115 doi:10.1186/s...
Saturday, November 11, 2017

RESONANCIA MAGNÉTICA CARDIACA EN ATAXIA DE FRIEDREICH: SEGUIMIENTO CARDIACO DE LAS TERAPIAS ANTIOXIDANTES

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Emilio Cuesta López. Tesis doctoral, UNIVERSIDAD AUTONOMA DE MADRID FACULTAD DE MEDICINA DEPARTAMENTO DE MEDICINA, Madrid 2017
Tuesday, November 7, 2017

Early cerebellar deficits in mitochondrial biogenesis and respiratory chain complexes in the KIKO mouse model of Friedreich ataxia

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Hong Lin, Jordi Magrane, Amy Rattelle, Anna Stepanova, Alexander Galkin, Elisia M. Clark, Yi Na Dong, Sarah M. Halawani, David R. Lynch; Dis...
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...