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...
Wednesday, February 22, 2017

E3 Ligase RNF126 Directly Ubiquitinates Frataxin, Promoting Its Degradation: Identification of a Potential Therapeutic Target for Friedreich Ataxia

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Monica Benini, Silvia Fortuni, Ivano Condò, Giulia Alfedi, Florence Malisan, Nicola Toschi, Dario Serio, Damiano Sergio Massaro, Gaetano Arc...
Thursday, February 16, 2017

A study of CTI-1601 for the treatment of Friedreich's Ataxia.

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Adis International Ltd. Part of Springer Science+Business Media, Trials Planning Phase of Trial: Phase I Latest Information Update: 14 ...
Tuesday, February 14, 2017

Health technology assessment of drugs for rare diseases: insights, trends, and reasons for negative recommendations from the CADTH common drug review

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Ghayath Janoudi, William Amegatse, Brendan McIntosh, Chander Sehgal, and Trevor Richter; Orphanet J Rare Dis. 2016; 11: 164. doi:10.1186/s13...
Saturday, February 11, 2017

Industrializing rare disease therapy discovery and development

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Sean Ekins; Nature Biotechnology 35, 117–118 (2017) doi:10.1038/nbt.3787 Published online 08 February 2017 There would also need to be clo...