Monday, December 7, 2015

Analyzing the Effects of a G137V Mutation in the FXN Gene

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Nathalie Faggianelli, Rita Puglisi, Liana Veneziano, Silvia Romano, Marina Frontali, Tommaso Vannocci, Silvia Fortuni, Roberto Testi and Ann...
Sunday, December 6, 2015

IFN-γ for Friedreich ataxia: present evidence.

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McKenzie Wells, Lauren Seyer, Kimberly Schadt & David R Lynch; Neurodegenerative Disease Management, Posted online on December 4, 2015, ...
Friday, December 4, 2015

Systematic review and clinical recommendations for dosage of supported home-based standing programs for adults with stroke, spinal cord injury and other neurological conditions

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Ginny Paleg and Roslyn Livingstone. BMC Musculoskelet Disord. 2015; 16: 358. Published online 2015 Nov 17. doi: 10.1186/s12891-015-0813-x ...
Wednesday, December 2, 2015

GIFT-1, a phase IIa clinical trial to test the safety and efficacy of IFNγ administration in FRDA patients.

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Christian Marcotulli, Silvia Fortuni, Gaetano Arcuri, Barbara Tomassini, Luca Leonardi, Francesco Pierelli, Roberto Testi, Carlo Casali; Neu...
Tuesday, December 1, 2015

Dr Evans–Galea, a leader in the field of gene therapy, talks about the challenges she has faced, but also the increasing visibility of female scientists in her field, and her professional mission: finding a cure for Friedrich ataxia

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www.theguardian.com, interview by Brigid Delaney, Tuesday 1 December The FXN gene was first discovered in the 90s, and there are potentia...

La phase de double appui : paramètre prédictif de la dégradation de la marche dans l’ataxie de Friedreich ?

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B. Roche, R. Martin, I. Husson, Neurophysiologie Clinique/Clinical Neurophysiology, Volume 45, Issues 4–5, November 2015, Pages 403-404, ISS...
Monday, November 30, 2015

Gene-editing tool CRISPR opens a world of possibilities for rare genetic diseases

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CRISPR gene-editing tool has scientists thrilled — but nervous. By Kelly Crowe, CBC News Posted: Nov 30, 2015 5:00 AM ET The CRISPR system...
Wednesday, November 25, 2015

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

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Gammie T, Lu CY, Babar ZU-D (2015), PLoS ONE 10(10): e0140002., doi:10.1371/journal.pone.0140002 OPEN ACCESS Access to orphan drugs depe...
Tuesday, November 24, 2015

Alternative mitochondrial electron transfer for the treatment of neurodegenerative diseases and cancers: Methylene blue connects the dots

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Shao-Hua Yang, Wenjun Li, Nathalie Sumien, Michael Forster, James W. Simpkins, Ran Liu, Progress in Neurobiology, Available online 18 Novemb...
Saturday, November 21, 2015

Retrotope announces opening of second clinical trial site for enrollment in Friedreich's ataxia clinical trial

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LOS ALTOS, Calif., Nov. 20, 2015 /PRNewswire/ -- Retrotope announces the opening of second clinical trial site, the Collaborative Neuroscien...