Friedreich Ataxia Scientific News

Friedreich Ataxia and close related scientific news. Topics related to rare diseases.

Wednesday, November 27, 2019

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT04176991. Chondrial Therapeutics, Inc., November 26, 2019 Phase 1, A Phase 1 Single Ascending Dose Study...
Monday, November 25, 2019

Mitochondrial dysfunction in neurons in Friedreich's ataxia

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Anna Stepanova, Jordi Magrané, Molecular and Cellular Neuroscience, 2019, 103419, doi:10.1016/j.mcn.2019.103419. Friedreich's ataxia i...
Saturday, November 23, 2019

Mitochondria Regulation in Ferroptosis

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Hai Wang, Can Liu, Yongxin Zhao, Ge Gao, European Journal of Cell Biology, 2019, doi:10.1016/j.ejcb.2019.151058. Ferroptosis is recognized...
Friday, November 22, 2019

TAK-831 for Friedreich Ataxia. Withdrawn from the Union Register of orphan medicinal products in November 2019

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20/11/2019. European Medicines Agency. On 1 April 2019, orphan designation (EU/3/19/2148) was granted by the European Commission to Takeda ...

N-terminomics/TAILS profiling of macrophages after chemical inhibition of legumain

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Bethany M AndersonLuiz G. N. de AlmeidaHenna SekhonDaniel YoungAntoine DufourLaura Edgington-Mitchell; Biochemistry 2019, XXXX, XXX, XXX-XXX...
Thursday, November 21, 2019

Analysis of Friedreich's ataxia patient clinical data reveals importance of accurate GAA repeat determination in disease prognosis and gender differences in cardiac measures

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Mohammadmersad Ghorbani, Françoise Pousset, Allan Tucker, Stephen Swift, Paola Giunti, Michael Parkinson, David Gilbert, XiaoHui Liu, Annett...
Wednesday, November 20, 2019

Metformin-dependent Exacerbation of Pathologies in Friedreich’s Ataxia

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Free Radical Biology and Medicine, Volume 145, Supplement 1, 2019, Page S81, doi:10.1016/j.freeradbiomed.2019.10.216. One case report desc...
Tuesday, November 19, 2019

Left ventricular structural and functional changes in Friedreich ataxia – Relationship with body size, sex, age and genetic severity

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Peverill RE, Romanelli G, Donelan L, Hassam R, Corben LA, Delatycki MB (2019); PLoS ONE 14(11): e0225147. doi:10.1371/journal.pone.0225147 ...
Tuesday, November 12, 2019

Minoryx Therapeutics receives Orphan Drug Designation from the European Commission for leriglitazone in the treatment of Friedreich’s Ataxia

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Mataró, Barcelona, Spain and Charleroi, Belgium, November 12, 2019 – Minoryx Therapeutics, a company that specializes in the development of ...
Monday, November 11, 2019

Reducing complexity: explaining inborn errors of metabolism and their treatment to children and adolescents

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Nina A. Zeltner, Mendy M. Welsink-Karssies, Markus A. Landolt, Dominique Bosshard-Bullinger, Fabia Keller, Annet M. Bosch, Marike Groenendij...
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BSc, MSc (Pharmacy), MSc (Computer science)
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