Thursday, June 4, 2020

Functional coupling of presequence processing and degradation in human mitochondria

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Kücükköse, C., Taskin, A.A., Marada, A., Brummer, T., Dennerlein, S. and Vögtle, F.‐N. (2020), FEBS J. doi:10.1111/febs.15358 The mitochon...
Friday, May 29, 2020

Significance of NT-proBNP and High-sensitivity Troponin in Friedreich Ataxia

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Legrand, L.; Maupain, C.; Monin, M.-L.; Ewenczyk, C.; Isnard, R.; Alkouri, R.; Durr, A.; Pousset, F.; J. Clin. Med. 2020, 9, 1630. doi:10.33...
Friday, May 22, 2020

Efficacy and Safety of CSF- Delivered AVXS-401 in Mice and NonHuman Primates for the Treatment of Friedreich’s Ataxia

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Martin Fugere, Rajeev Sivasankaran, Susan McQuown, Chang Choi, Katrina Salvador, Shirley Phillips, Yin Gu, Binh Chu, Janet Do, Tsun-Kai Chan...
Thursday, May 21, 2020

PTC announced delays in its gene therapy program for Friedreich’s ataxia

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MAY 19, 2020. PTC announced delays in its gene therapy program for Friedreich’s ataxia and in the opening of a manufacturing facility in Ne...

Minoryx Therapeutics doses first patient with leriglitazone in registration-enabling cALD NEXUS trial

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20-May-2020. PharmiWeb.com. Mataró, Barcelona, Spain and Charleroi, Belgium, May 12, 2020 – Minoryx Therapeutics, a company that specializes...
Sunday, May 17, 2020

Non-Invasive Cerebellar Stimulation in Neurodegenerative Ataxia: A Literature Review

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Benussi A, Pascual-Leone A, Borroni B.; International Journal of Molecular Sciences. 2020 Mar;21(6). DOI: 10.3390/ijms21061948. In light o...
Wednesday, May 13, 2020

J. Analysis of Putative Epigenetic Regulatory Elements in the FXN Genomic Locus

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Fernández-Frías, I.; Pérez-Luz, S.; Díaz-Nido. Int. J. Mol. Sci. 2020, 21, 3410. Friedreich´s ataxia (FRDA) is an autosomal recessive dise...
Sunday, May 10, 2020

Neurofilament light chain as a potential biomarker of disease status in Friedreich ataxia

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Alexandra Clay, Kristin M. Obrochta, Russell K. Soon Jr, Christopher B. Russell & David R. Lynch; Neurol (2020). doi:10.1007/s00415-020...
Tuesday, May 5, 2020

Therapeutic potential of stem cells for treatment of neurodegenerative diseases

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Ahmadian-Moghadam, H., Sadat-Shirazi, M. & Zarrindast, M.; Biotechnol Lett (2020). doi:10.1007/s10529-020-02886-1 The regenerative pot...
Monday, May 4, 2020

CRISPR/Cas9 gene editing of hematopoietic stem cells from patients with Friedreich’s ataxia

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Celine J. Rocca, Joseph N. Rainaldi, Jay Sharma, Yanmeng Shi, Joseph H. Haquang, Jens Luebeck, Prashant Mali, Stephanie Cherqui, Molecular T...