Friday, June 2, 2017

Is Reata's Friedreich's Ataxia Study a Failure?

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Rare Disease ReportData from a Phase 2 Friedreich’s ataxia (FA) study by Reata Pharmaceuticals is making its rounds on social media today. T...

Reata Pharmaceuticals, Inc. Announces Positive Data From Part One of Moxie Trial of Omaveloxolone for Friedreich’s Ataxia

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IRVING, Texas, June 01, 2017 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq:RETA) (“Reata” or “the Company”), a clinical-stage biop...

Friedreich’s ataxia induced pluripotent stem cell-derived cardiomyocytes display electrophysiological abnormalities and calcium handling deficiency

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Duncan E. Crombie, Claire L. Curl, Antonia JA Raaijmakers, Priyadharshini Sivakumaran, Tejal Kulkarni, Raymond CB Wong, Itsunari Minami, Mar...
Thursday, June 1, 2017

Single-step blood direct PCR: A robust and rapid method to diagnose triplet repeat disorders

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Inder Singh, Vishnu Swarup, Sunil Shakya, Vinay Goyal, Mohammed Faruq, Achal Kumar Srivastava, Single-step blood direct PCR: A robust and ra...
Wednesday, May 31, 2017

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry

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F Mavilio; Gene Therapy , (25 May 2017) doi:10.1038/gt.2017.36 For the last 20 years, academic research has been the major, and often onl...
Tuesday, May 30, 2017

Blood–brain barrier peptide shuttles

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Macarena Sánchez-Navarro, Ernest Giralt, Meritxell Teixidó, Current Opinion in Chemical Biology, Volume 38, June 2017, Pages 134-140, ISSN 1...
Monday, May 29, 2017

Cerebral compensation during motor function in Friedreich ataxia: The IMAGE-FRDA study

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Harding, I. H., Corben, L. A., Delatycki, M. B., Stagnitti, M. R., Storey, E., Egan, G. F. and Georgiou-Karistianis, N. (2017), Mov. Disord...
Sunday, May 28, 2017

In Vitro Antioxidant Activity of Idebenone Derivative-Loaded Solid Lipid Nanoparticles

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Lucia Montenegro, Maria N. Modica, Loredana Salerno, Anna Maria Panico, Lucia Crascì, Giovanni Puglisi and Giuseppe Romeo; Molecules 2017, 2...
Saturday, May 27, 2017

Structural signature of classical versus late-onset friedreich's ataxia by Multimodality brain MRI

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Thiago Junqueira R. Rezende, Alberto Rolim M. Martinez, Ingrid Faber, Karen Girotto, José Luiz Pedroso, Orlando G. Barsottini, Iscia Lopes-C...
Friday, May 26, 2017

Progressive mitochondrial protein lysine acetylation and heart failure in a model of Friedreich’s ataxia cardiomyopathy

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Stram AR, Wagner GR, Fogler BD, Pride PM, Hirschey MD, Payne RM (2017). PLoS ONE 12(5): e0178354. doi:10.1371/journal.pone.0178354 Acetyla...