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...
Friday, February 10, 2017

Mitochondria-penetrating peptides conjugated to desferrioxamine as chelators for mitochondrial labile iron

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Alta RYP, Vitorino HA, Goswami D, Liria CW, Wisnovsky SP, Kelley SO, Machini MT, Espósito BP. (2017). PLoS ONE 12(2): e0171729. doi:10.1371/...
Thursday, February 9, 2017

Shire divests mRNA platform to RaNa

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Nature Biotechnology 35,103 (2017) doi:10.1038/nbt0217-103b Published online 08 February 2017 The Shire deal includes preclinical drug pro...
Wednesday, February 8, 2017

Comparison of Two Different PCR-based Methods for Detection of GAA Expansions in Frataxin Gene

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Mona ENTEZA­M, Akbar AMIRFIROOZI, Mansoureh TOGHA, Mohammad KERAMATIPOUR; Iranian Journal of Public Health 2017. 46(2):222-228. The presen...
Tuesday, February 7, 2017

Chondrial Therapeutics Secures Up to $22.6 Million in Series A Financing and Licenses Novel Technology for the Treatment of Friedreich's Ataxia

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PR Newswire, News provided by Chondrial Therapeutics, Inc. Feb 06, 2017, Company Plans to Advance Pipeline of Product Candidates Focused on...
Friday, February 3, 2017

MiRNA-145 Regulates the Development of Congenital Heart Disease Through Targeting FXN

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Lei Wang, Danqiu Tian, Jihua Hu, Haijian Xing, Min Sun, Juanli Wang, Qiang Jian, Hua Yang. Pediatr Cardiol (2016) 37: 629. doi:10.1007/s0024...
Thursday, February 2, 2017

Estudio piloto sobre los valores acústicos de las vocales en español como indicadores de la gravedad de la disartria

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Pilot study of the acoustic values of the vowels in Spanish as indicators of the severity of dysarthria. (Article in Spanish; Abstract avail...