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...
Saturday, May 29, 2021

Mitochondrial and metabolic dysfunction in Friedreich ataxia: update on pathophysiological relevance and clinical interventions

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David R. Lynch, Garrett Farmer; Mitochondrial and metabolic dysfunction in Friedreich ataxia: update on pathophysiological relevance and cli...
Friday, May 28, 2021

The Oxford-Harrington Rare Disease Centre initiates first disease priority area: Friedreich’s Ataxia

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27 May 2021, The Oxford-Harrington Rare Disease Centre is redoubling efforts to develop a therapeutics programme for Friedreich’s Ataxia. OH...
Thursday, May 27, 2021

Iron-sulfur cluster deficiency can be sensed by IRP2 and regulates iron homeostasis and sensitivity to ferroptosis independent of IRP1 and FBXL5

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ERDEM M. TERZI, VLADISLAV O. SVIDERSKIY, SAMANTHA W. ALVAREZ, GABRIELLE C. WHITEN, RICHARD POSSEMATO; Science Advances 26 May 2021: Vol. 7,...
Wednesday, May 26, 2021

Larimar Therapeutics Reports FDA Clinical Hold on CTI-1601 and Termination of Recently Announced Private Placement Financing

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BALA CYNWYD, Pa., May 25, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology com...
Tuesday, May 25, 2021

Generation of a Friedreich’s Ataxia patient-derived iPSC line USFi001-A

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Mariana Burgos Angulo, Jiajia Yang, Mariana A. Argenziano, Alexander C. Bertalovitz, Maliheh Najari Beidokhti, Thomas V. McDonald; Stem Cell...
Sunday, May 23, 2021

Larimar Therapeutics Announces $95 Million Private Placement Financing

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BALA CYNWYD, Pa., May 21, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company f...