Monday, January 11, 2021

Jupiter Orphan Therapeutics Announces First Patient Dosed in Phase I Dose Escalation Study of JOTROL™

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JUPITER, Fla., Jan. 11, 2021 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc. ("JOT") a biotechnology company pioneering a novel ...

Cytoprotective activities of kinetin purine isosteres

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Barbara Maková, Václav Mik, Barbora Lišková, Gabriel González, Dominik Vítek, Martina Medvedíková, Beata Monfort, Veronika Ručilová, Alena K...

DNA REPAIR PATHWAYS ARE ALTERED IN NEURAL CELL MODELS OF FRATAXIN DEFICIENCY

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Jara Moreno-Lorite, Sara Pérez-Luz, Yurika Katsu-Jiménez, Daniel Oberdoerfer, Javier Díaz-Nido, Molecular and Cellular Neuroscience, 2021, 1...
Friday, January 8, 2021

Friedreich ataxia in COVID-19 time: current impact and future possibilities

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Tommaso Schirinzi, Andrea Sancesario, Enrico Castelli, Enrico Bertini & Gessica Vasco; Cerebellum & Ataxias volume 8, Article number...

Current report, slide presentation, Form 8-K Exicure, Inc.

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Form 8-K Exicure, Inc. Current report, items 7.01 and 9.01; Published: 2021-01-07 On January 7, 2021, Exicure, Inc. (the “Company”) will ho...

LEXEO Therapeutics Launches with $85 Million Series A Financing to Develop Gene Therapies for Rare and Non-Rare Monogenic Diseases

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NEW YORK, Jan. 07, 2021 (GLOBE NEWSWIRE) -- LEXEO Therapeutics, a clinical-stage gene therapy company, debuted today with an oversubscribed ...
Wednesday, January 6, 2021

Developing an Instrumented Measure of Upper Limb Function in Friedreich Ataxia

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Louise A. Corben, Khoa D. Nguyen, Pubudu N. Pathirana, Malcolm K. Horne, David J. Szmulewicz, Melissa Roberts & Martin B. Delatycki; Ce...
Tuesday, January 5, 2021

Two ways to control CjCas9 expression in the deletion of pathogenic GAA repeat in frataxin gene

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Pouiré Yaméogo; Genetic Engineering Research, Vol.9 Iss.3:sc165 Our results showed that despite the self-destruction of the CjCas9 gene, an ...
Thursday, December 31, 2020

Antisense Transcription across Nucleotide Repeat Expansions in Neurodegenerative and Neuromuscular Diseases: Progress and Mysteries

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Castro AF, Loureiro JR, Bessa J, Silveira I. Genes. 2020 Nov;11(12). DOI: 10.3390/genes11121418.

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich's Ataxia: Retrospective Study

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Mehmet Fatih YETKİN and Murat GÜLTEKİN; Noro Psikiyatr Ars. 2020 Sep 21;57(4):270-273. doi: 10.29399/npa.25047. eCollection 2020 Dec.   Inte...