Friday, March 17, 2017

A wearable proprioceptive stabilizer for rehabilitation of limb and gait ataxia in hereditary cerebellar ataxias: a pilot open-labeled study

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Luca Leonardi, Maria Gabriella Aceto, Christian Marcotulli, Giuseppe Arcuria, Mariano Serrao, Francesco Pierelli, Paolo Paone, Alessandro Fi...
Thursday, March 16, 2017

Voyager Therapeutics: Advance multiple preclinical programs towards clinical trials, with the goal of filing three IND applications within the next 24-months VY-FXN01 program

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Voyager Therapeutics Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Highlights. CAMBRIDGE, Mass., March 15, 2017 ...
Tuesday, March 14, 2017

Affordable orphan drugs: a role for not-for-profit organizations

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Davies, E. H., Fulton, E., Brook, D., and Hughes, D. A. (2017). Br J Clin Pharmacol, doi: 10.1111/bcp.13240. Although the regulatory steps...
Monday, March 13, 2017

Early-Onset Friedreich's Ataxia With Oculomotor Apraxia

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Amene Saghazadeh, Sina Hafizi, Firouzeh Hosseini, Mahmoud Reza Ashrafi, Nima Rezaei; Acta Medica Iranica 2017. 55(2):128-130. Here we desc...
Sunday, March 12, 2017

Tolérance à l’exercice et capacité physique des patients atteints d’une ataxie de Friedreich tardive

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Médéric Descoins, Chantal Verkindt, Bruno Lemarchand, Dalleau Georges, Nicolas Perrot, Claude Mignard, Ariane Choumert, Revue Neurologique, ...
Saturday, March 11, 2017

Frataxin-Deficiency in the Heart Results in an Impaired Nrf2 Response: A Dual Mechanism Mediated via Up-Regulation of Keap1 and GSK3β Axis

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Amy Anzovino, Shannon Chiang, Bronwyn E Brown, Clare L Hawkins, Des R Richardson, Michael LH Huang, Free Radical Biology and Medicine, Volum...
Friday, March 10, 2017

A role for astrocytes in cerebellar deficits in frataxin deficiency: Protection by insulin-like growth factor I

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C Franco, L Genis, JA Navarro, P Perez-Domper, AM Fernandez, S Schneuwly, I Torres Alemán, Molecular and Cellular Neuroscience, Available on...
Thursday, March 9, 2017

Pfizer looks at building major gene therapy manufacturing facility in North Carolina

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FiercePharma.  Eric Palmer, Mar 8, 2017. Pfizer, which scooped up Bamboo Therapeutics last year in its aim to be a major player in gene the...
Thursday, March 2, 2017

Iron Accumulation, Glutathione Depletion, and Lipid Peroxidation Must Occur Simultaneously during Ferroptosis and are Mutually Amplifying Events

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Robert L. Bertrand, Medical Hypotheses, Available online 28 February 2017, ISSN 0306-9877, doi:10.1016/j.mehy.2017.02.017. It is proposed ...
Friday, February 24, 2017

Measuring Inhibition and Cognitive Flexibility in Friedreich Ataxia

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Louise A. Corben, Felicity Klopper, Monique Stagnitti, Nellie Georgiou-KaristianisJohn L. Bradshaw, Gary RanceMartin B. Delatycki; Cerebell...