Tuesday, May 23, 2023

PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial Twitter Facebook LinkedIn GooglePlus Pinterest

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SOUTH PLAINFIELD, N.J. , May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA ...

Large-scale expansions of Friedreich's ataxia GAA•TTC repeats in an experimental human system: role of DNA replication and prevention by LNA-DNA oligonucleotides and PNA oligomers

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Anastasia Rastokina, Jorge Cebrián, Negin Mozafari, Nicholas H Mandel, C I Edvard Smith, Massimo Lopes, Rula Zain, Sergei M Mirkin, Large-sc...
Monday, May 22, 2023

The Loss of Frataxin Impairs Microglia Homeostatic Functions in Friedreich’s Ataxia

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Ferrara G. Abstracts of the Fifth Brainstorming Research Assembly for Young Neuroscientists (BraYn), Italy, 28-30 September 2022. Neurology ...
Sunday, May 21, 2023

Neuropsychiatric symptoms in spinocerebellar ataxias and Friedreich ataxia

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Karamazovova S, Matuskova V, Ismail Z, Vyhnalek M. Neuropsychiatric symptoms in spinocerebellar ataxias and Friedreich ataxia. Neuroscience ...
Wednesday, May 17, 2023

Patient-Derived iPSC-Neurons and Microglia for Modeling Friedreich’s Ataxia Disease

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1694 Patient-Derived iPSC-Neurons and Microglia for Modeling Friedreich’s Ataxia Disease; Priyanka Mishra, Anusha Sivakumar, Avalon Johnson,...

Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia

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1656 Safety of Ascending Doses of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich’s Ataxia; Jonathan B. Rosenberg, Alessandri...

Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications

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1549 Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications; Jennifer C. G. Green, Jessica F. Boehl...

Comparison of Cardiac Specific Promoters to Liver-Specific miRNA Targets to Maximize Cardiac vs Liver Expression Following Intravenous AAVrh.10-Mediated Cardiac Gene Therapy

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1518 Comparison of Cardiac Specific Promoters to Liver-Specific miRNA Targets to Maximize Cardiac vs Liver Expression Following Intravenous ...

CRISPR/Cas9 Edited Hematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia

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1354 CRISPR/Cas9 Edited Hematopoietic Stem and Progenitor Cells for Friedreich’s Ataxia; Anusha Sivakumar, Rafael Andres Badell-Grau, Rita W...

Reversal of Cardiac Phenotype in a Mouse Model of Friedreich’s Ataxia Following Administration of AAV Gene Therapy

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1184 Reversal of Cardiac Phenotype in a Mouse Model of Friedreich’s Ataxia Following Administration of AAV Gene Therapy; Joshua C. Chang, Wh...