Monday, June 7, 2021

Quantitative Assessment of Friedreich Ataxia via Self-Drinking Activity

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R. Krishna, P. N. Pathirana, M. K. Horne, L. A. Corben and D. J. Szmulewicz; IEEE Journal of Biomedical and Health Informatics, vol. 25, no....
Sunday, June 6, 2021

Coexistence of Tyrosinemia and Friedreich Ataxia in a Single Patient: Treatment with Liver Transplantation

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Diya Cherian, Kimberly Schadt, Courtney Park, Stephanie Veasey, David Goldberg, David Lynch; Ann Case Report 6: 581. DOI: 10.29011/2574-7754...
Saturday, June 5, 2021

Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery

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Application US16/651,617- Assigned to VOYAGER THERAPEUTICS, INC.  Described herein are compositions and methods for treating Friedreich'...
Friday, June 4, 2021

AavantiBio lands manufacturing partner in Friedreich's Ataxia program; NC Research Triangle lands another CDMO

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June 3, 2021. ENDPOINTNEWS. AavantiBio and Resilience have announced a collaboration to manufacture a pipeline of therapies, including Aavan...
Thursday, June 3, 2021

FARA FA Pipeline Webinar - Jun 2, 2021

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Molecular Details of the Frataxin–Scaffold Interaction during Mitochondrial Fe–S Cluster Assembly

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Campbell, C.J.; Pall, A.E.; Naik, A.R.; Thompson, L.N.; Stemmler, T.L.; Int. J. Mol. Sci. 2021, 22, 6006. doi:10.3390/ijms22116006 Molecula...
Tuesday, June 1, 2021

Results of a randomized double-blind study evaluating luvadaxistat in adults with Friedreich ataxia

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Hao Wang, Jonathan Norton, Lin Xu, Nicholas DeMartinis, Rohini Sen, Ankit Shah, Jennifer Farmer, David Lynch; Annals of Clinical and Transl...
Monday, May 31, 2021

SAFETY AND EFFICACY OF THE STEM CELL TRANSPLANTATION IN FRIEDREICH’S ATAXIA: A REPORT OF THREE CASES

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Riza Azeri, Duygu Koyuncu Irmak, Eda Sun, Erdal Karaöz; Int J Physiother. Vol 8(1), 31-35, February (2021) doi:10.15621/ijphy/2021/v8i1/903 ...
Sunday, May 30, 2021

An unusual combination of large Eustachian valve in a young patient with Friedreich's ataxia cardiomyopathy

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Stylianos A. Karvounaris, Georgios S. Papaetis, Petros P. Mavrommatis; Cardiol J 2021;28(3):498-499. DOI: 10.5603/CJ.2021.0051 According to...

Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders

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Odiba AS, Okoro NO, Durojaye OA, Wu Y., Open Life Sciences. 2021 ;16(1):431-441. DOI: 10.1515/biol-2021-0033. Molecular biology and biotechn...