Friday, April 12, 2019

Mechanism of frataxin “bypass” in human iron-sulfur cluster biosynthesis with implications for Friedreich’s ataxia

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Deepika Das, Shachin Patra, Jennifer Bridwell-Rabb, and David P. Barondeau; J. Biol. Chem. jbc.RA119.007716. doi:10.1074/jbc.RA119.007716 ...
Tuesday, April 2, 2019

L’ataxie de Friedreich : la particularité subsaharienne

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Ale Thiam, Amadou Koura Ndao, Mansour Ndiaye, Fatou Diouf Sene, Amadou Gallo Diop, Ibrahima Pierre Ndiaye, Astou Thiam, Revue Neurologique, ...
Saturday, March 30, 2019

StrideBio and Takeda Enter Collaboration and License Agreement to Advance Novel Gene Therapies for Neurological Diseases

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DURHAM, N.C., March 28, 2019 /PRNewswire/ — StrideBio, Inc, a leading developer of novel adeno-associated viral (AAV) based gene therapies, ...
Thursday, March 28, 2019

New Insights into the Hepcidin-Ferroportin Axis and Iron Homeostasis in iPSC-Derived Cardiomyocytes from Friedreich’s Ataxia Patient

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Alessandra Bolotta, Provvidenza Maria Abruzzo, Vito Antonio Baldassarro, Alessandro Ghezzo, Katia Scotlandi, Marina Marini and Cinzia Zucchi...
Tuesday, March 26, 2019

Open Trail of γIFN for Friedreich Ataxia

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ClinicalTrials.gov Identifier: NCT03888664. IRCCS Eugenio Medea The investigator proposes an open label pilot study to investigate the saf...
Monday, March 25, 2019

Therapeutic Prospects for Friedreich’s Ataxia

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Siyuan Zhang, Marek Napierala, Jill S. Napierala; Trends in Pharmacological Sciences, Volume 40, Issue 4, 2019, Pages 229-233, doi:10.1016/j...
Thursday, March 21, 2019

Ion Mobility-Mass Spectrometry Reveals Details of Formation and Structure for GAA·TCC DNA and RNA Triplexes

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Jiawei Li, Alexander Begbie, Belinda J. Boehm, Alexander Button, Charles Whidborne, Yannii Pouferis, David M. Huang, Tara L. Pukala; J. Am. ...
Wednesday, March 20, 2019

Patient reported outcomes in Friedreich's Ataxia after withdrawal from Idebenone

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Cook, A. , Boesch, S. , Heck, S. , Brunt, E. , Klockgether, T. , Schöls, L. , Schulz, A. and Giunti, P. (2019). Acta Neurol Scand. Accepted ...
Tuesday, March 19, 2019

CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review.

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Babačić H, Mehta A, Merkel O, Schoser B (2019); PLOS ONE 14(2): e0212198. doi:10.1371/journal.pone.0212198 Here we give a systematic summa...
Saturday, March 16, 2019

Inherited Ataxia and Intrathecal Baclofen for the Treatment of Spasticity and Painful Spasms

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Berntsson S, G, Gauffin H, Melberg A, Holtz A, Landtblom A, M; Stereotact Funct Neurosurg 2019. doi: 10.1159/000497165 Intrathecal baclof...