Wednesday, December 7, 2016

Researchers uncover possible source of genetic error causing multiple diseases

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Phys.org, Tufts University, December 5, 2016. Tufts University researchers have discovered a possible explanation for the occurrence of a ...
Tuesday, December 6, 2016

Protective role of frataxin against myocardial ischemia reperfusion injury

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Abdullah Al Asmari; Global Summit on Heart Diseases and Therapeutics, October 20-21, 2016 Chicago, USA. Posters & Accepted Abstracts: J ...
Monday, December 5, 2016

Antibióticos y trastornos de la marcha [Antibiotics and gait disorders]

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Gómez-Porro P, Vinagre-Aragón A, Zabala-Goiburu JA. Rev Neurol 2016; 63: 501-9. [Article in Spanish] Revisión La toxicidad neurológica d...
Sunday, December 4, 2016

Loss of Frataxin activates the iron/sphingolipid/PDK1/Mef2 pathway in mammals

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Kuchuan Chen, Tammy Szu-Yu Ho, Guang Lin, Kai Li Tan, Matthew N Rasband, Hugo J Bellen; eLife Tue, 29 Nov 2016 DOI:10.7554/eLife.20732 Here...
Friday, December 2, 2016

Negotiating prices of drugs for rare diseases

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Séverine Henrard & Francis Arickx, Bulletin of the World Health Organization 2016;94:779-781. doi:10.2471/BLT.15.163519 Due to the in...

A Cost of Illness Study Evaluating The Healthcare And Societal Burden of Friedreich’s Ataxia In The United Kingdom

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K Hanman, A Griffiths, A Bobrowska, J Vallortigara, J Greenfield, RS Thompson, Value in Health, Volume 19, Issue 7, November 2016, Pages A58...
Thursday, December 1, 2016

Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase

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Cinzia Cinesi, Lorène Aeschbach, Bin Yang & Vincent Dion. Nature Communications 7, Article number: 13272 (2016) doi:10.1038/ncomms13272 ...
Wednesday, November 30, 2016

Launches of First Therapies Approved for Spinal Muscular Atrophy and Friedreich's Ataxia Will Revolutionize Treatment and Drive Growth of These Rare Disease Markets

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BURLINGTON, Mass., Oct. 25, 2016 /PRNewswire/ -- Decision Resources Group finds that the anticipated launches of the first therapies for the...

Disruption of Higher Order DNA Structures in Friedreich’s Ataxia (GAA)n Repeats by PNA or LNA Targeting

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Helen Bergquist, Cristina S. J. Rocha, Rubén Álvarez-Asencio, Chi-Hung Nguyen, Mark. W. Rutland, C. I. Edvard Smith, Liam Good, Peter E. Nie...
Tuesday, November 29, 2016

Long term clinical and neurophysiological effects of cerebellar transcranial direct current stimulation in patients with neurodegenerative ataxia

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A. Benussi, V. Dell’Era, M.S. Cotelli, M. Turla, C. Casali, A. Padovani, B. Borroni, Brain Stimulation, Available online 3 November 2016, IS...