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...
Thursday, November 19, 2015

Frataxin expression in reticulocytes of non-splenectomized and splenectomized patients with HbE-β-thalassaemia

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Yollada Suebpeng, Arunee Jetsrisuparb, Supan Fucharoen, Amporn Tripatara, Clinical Biochemistry, Available online 14 November 2015, ISSN 000...
Wednesday, November 18, 2015

Efficient attenuation of Friedreich's ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA,

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Yee-Ki Lee, Yee-Man Lau, Kwong-Man Ng, Wing-Hon Lai, Shu-Leong Ho, Hung-Fat Tse, Chung-Wah Siu, Philip Wing-Lok Ho, International Journal o...
Saturday, November 14, 2015

Life without Fe-S clusters

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Agostinho G. Rocha and Andrew Dancis, Molecular Microbiology 1365-2958, DOI - 10.1111/mmi.13273 Microcommentary on “Novel features of the ...