Friedreich Ataxia Scientific News
Friedreich Ataxia and close related scientific news. Topics related to rare diseases.
Saturday, July 22, 2023
Understanding the Impact of Entrada Therapeutics Inc. on NASDAQ:TRDA
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JUL 22, 2023. CORP THESPYWHOBILLEDME. The company’s innovative approach to drug development has been a key factor in its success. Entrada Th...
Friday, July 21, 2023
Patient Dosing Complete for Part 1 of Gene Therapy Trial in Friedreich Ataxia Cardiomyopathy
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Neurologylive; Jul 20, 2023. According to a recent announcement, patient dosing in the first cohort of the SUNRISE-FA study (NCT05445323), ...
Tuesday, July 11, 2023
Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy
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Ian Blair, Teerapat Rojsajjakul, Juliette Hordeaux et al. Quantification of human mature frataxin protein expression in nonhuman primate hea...
PPAR-gamma agonist pioglitazone recovers mitochondrial quality controls in fibroblasts from PITRM1-deficient patients
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DArio Brunetti, A. D., Donfrancesco, C., Berlingieri, C., Frascarelli, M., Giacomello, A. P., Magalhaes Rebelo, L., Bindoff, S., Reeval, P.,...
Monday, July 10, 2023
Non-B DNA structures as a booster of genome instability
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Renée C. Duardo, Federico Guerra, Simona Pepe, Giovanni Capranico, Non-B DNA structures as a booster of genome instability, Biochimie, 2023,...
Targeting Ion Channels and Purkinje Neuron Intrinsic Membrane Excitability as a Therapeutic Strategy for Cerebellar Ataxia
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Huang H, Shakkottai VG. Targeting Ion Channels and Purkinje Neuron Intrinsic Membrane Excitability as a Therapeutic Strategy for Cerebellar ...
Novel Therapeutic Approaches in Inherited Neuropathies: A Systematic Review
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Hustinx M, Shorrocks AM, Servais L. Novel Therapeutic Approaches in Inherited Neuropathies: A Systematic Review. Pharmaceutics. 2023 May;15(...
Direct Cysteine Desulfurase Activity Determination by NMR and the Study of the Functional Role of Key Structural Elements of Human NFS1
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Sewell KE, Gola GF, Pignataro MF, et al. Direct Cysteine Desulfurase Activity Determination by NMR and the Study of the Functional Role of K...
Tuesday, July 4, 2023
Alpha-tocopherylquinone differentially modulates claudins to enhance intestinal epithelial tight junction barrier via AhR and Nrf2 pathways
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Ashwinkumar Subramenium Ganapathy, Kushal Saha, Alexandra Wang, Priya Arumugam, Viszwapriya Dharmaprakash, Gregory Yochum, Walter Koltun, Me...
Monday, July 3, 2023
AI-based tools for the diagnosis and treatment of rare neurological disorders
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Molnar, M.J., Molnar, V. AI-based tools for the diagnosis and treatment of rare neurological disorders. Nat Rev Neurol (2023). doi:10.1038/s...
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