Thursday, January 30, 2025

'Editorial: The Mechanistic Investigation and Emerging Therapies for Friedreich's Ataxia'

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Yina Dong, Vijayendran Chandran, Elisabetta Soragni, David R Lynch, Front. Pharmacol. Sec. Neuropharmacology Volume 16 - 2025 doi:10.3389/fp...
Saturday, January 25, 2025

Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia

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BALA CYNWYD, Pa., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), today announced that dosing of adol...
Friday, January 24, 2025

Hypoxia as a medicine

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Robert S. Rogers, Vamsi K. Mootha. Hypoxia as a medicine. Science Translational Medicine 22 Jan 2025 Vol 17, Issue 782 DOI: 10.1126/scitrans...
Wednesday, January 22, 2025

Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

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CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that it has received Fast Track designation fro...
Monday, January 20, 2025

Friedreich ataxia: what can we learn from non-GAA repeat mutations?

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Lynch DR, Shen M, Wilson RB. Friedreich ataxia: what can we learn from non-GAA repeat mutations? Neurodegener Dis Manag. 2025 Jan 15:1-10. d...

Altered Intracerebellar Functional Connectivity in Friedreich's Ataxia: A Graph-Theory Functional MRI Study

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Tranfa M, Costabile T, Pontillo G, Scaravilli A, Pane C, Brunetti A, Saccà F, Cocozza S. Altered Intracerebellar Functional Connectivity in ...
Tuesday, January 14, 2025

Life and death of Yfh1: how cool is cold denaturation

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Temussi PA, Martin SR, Pastore A. Life and death of Yfh1: how cool is cold denaturation. Q Rev Biophys. 2025 Jan 13;58:e2. doi: 10.1017/S003...
Monday, January 13, 2025

Longitudinal analysis of anthropometric measures over 5 years in patients with Friedreich ataxia in the EFACTS natural history study

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Lischewski SA, Konrad K, Dogan I, Didszun C, Costa AS, Schawohl SA, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesc...
Friday, January 10, 2025

Effects of physiotherapy on degenerative cerebellar ataxia: a systematic review and meta-analysis

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Matsugi A, Bando K, Kondo Y, Kikuchi Y, Miyata K, Hiramatsu Y, Yamanaka Y, Tanaka H, Okuda Y, Haruyama K and Yamasaki Y (2025) Effects of ph...

Poincaré plot analysis of ECG uncovers beneficial effects of omaveloxolone in a mouse model of Friedreich’s ataxia

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Poincaré plot analysis of ECG uncovers beneficial effects of omaveloxolone in a mouse model of Friedreich’s ataxia, Figueroa, Francisco et a...