Friday, December 9, 2016

Horizon slumps after phase 3 Friedreich's ataxia trial flops

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FierceBiotech. by Nick Paul Taylor | Dec 8, 2016 9:40am. Horizon Pharma’s Actimmune has missed the primary endpoint in a phase 3 trial, spar...
Thursday, December 8, 2016

Horizon Pharma plc Announces Topline Results from Phase 3 Study of ACTIMMUNE® (interferon gamma-1b) in Friedreich's Ataxia

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DUBLIN, Ireland, Dec. 08, 2016 (GLOBE NEWSWIRE) -- Horizon Pharma plc (NASDAQ:HZNP), today announced that the Phase 3 trial, STEADFAST (Safe...
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...