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...
Friday, March 15, 2019

Assessment of cell-free levels of iron and copper in patients with Friedreich’s ataxia

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Deepti PathakAchal Kumar SrivastavaSheffali GulatiMoganty R. Rajeswari; Biometals (2019). https://doi.org/10.1007/s10534-019-00186-4 The i...
Wednesday, March 13, 2019

The role of robotic gait training and tDCS in Friedrich ataxia rehabilitation: A case report

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Simona Portaro;Margherita Russo;Alessia Bramanti;Antonio Leo;Luana Billeri;Alfredo Manuli;Gianluca La Rosa;Antonino Naro;Rocco Calabrò; Medi...