Saturday, July 1, 2023

Decreased filamentous actin and tight junction protein expression, and paracellular permeability in Frataxin-deficient human brain microvascular endothelial cells – implications for blood-brain barrier integrity in Friedreich's Ataxia

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Smith FM, Kosman DJ. Decreased filamentous actin and tight junction protein expression, and paracellular permeability in Frataxin-deficient ...

Frataxin inhibits the sensitivity of the myocardium to ferroptosis by regulating iron homeostasis

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Zhang Z, Jiang W, Zhang C, Yin Y, Mu N, Wang Y, Yu L, Ma H. Frataxin inhibits the sensitivity of the myocardium to ferroptosis by regulating...

Multiway sparse distance weighted discrimination

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Guo B, Eberly LE, Henry PG, Lenglet C, Lock EF. Multiway sparse distance weighted discrimination. J Comput Graph Stat. 2023;32(2):730-743. d...

Adult-Onset Neuroepidemiology in Finland: Lessons to Learn and Work to Do

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Sipilä JOT. Adult-Onset Neuroepidemiology in Finland: Lessons to Learn and Work to Do. Journal of Clinical Medicine. 2023 Jun;12(12):3972. D...
Thursday, June 29, 2023

Reata Pharmaceuticals Announces Approval of Prior Approval Supplement for SKYCLARYS® (Omaveloxolone) and Commercial Availability of SKYCLARYS for Patients with Friedreich’s Ataxia

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June 27, 2023. PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a ...
Monday, June 19, 2023

Retinal hypoplasia and degeneration result in vision loss in Friedreich ataxia

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Rodden, L.N., McIntyre, K., Keita, M., Wells, M., Park, C., Profeta, V., Waldman, A., Rummey, C., Balcer, L.J. and Lynch, D.R. (2023), Retin...
Friday, June 16, 2023

Anodal Cerebellar Transcranial Direct Current Stimulation Reduces Motor and Cognitive Symptoms in Friedreich's Ataxia: A Randomized, Sham-Controlled Trial

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Naeije, G., Rovai, A., Destrebecq, V., Trotta, N. and De Tiège, X. (2023), Anodal Cerebellar Transcranial Direct Current Stimulation Reduces...
Tuesday, June 13, 2023

LEXEO Therapeutics Announces Completion of First Cohort and Dosing in Second Cohort in SUNRISE-FA, a Phase 1/2 Clinical Trial of LX2006 for the Treatment of Friedreich’s Ataxia Cardiomyopathy

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NEW YORK, June 13, 2023 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated vir...

A Retrospective Claims Analysis Characterizing Health Care Resource Use Among Patients with Friedreich Ataxia in the United States,

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C. Qian, D. Lynch, L. Powell, A. Salvucci, G. Vasco, K.M Johnston, I. Tomazos, A Retrospective Claims Analysis Characterizing Health Care Re...

The Clinical Burden of Friedreich Ataxia: A Retrospective Claims Analysis in the United States

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L. Powell, D. Lynch, C. Qian, A. Salvucci, G. Vasco, K.M Johnston, I. Tomazos, The Clinical Burden of Friedreich Ataxia: A Retrospective Cla...