Thursday, March 26, 2026

Generation of Friedreich's ataxia induced pluripotent stem cells carrying the FXN c.165 + 5G>C splicing mutation

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Yameogo P, Gerhart BJ, Sentmanat MF, Neilson A, Cui X, Verma M, Lynch DR, Napierala JS, Napierala M. Generation of Friedreich's ataxia i...

Deciphering the missing links between Friedreich ataxia and multiple sclerosis for targeted drug development

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Kwa FAA, Anjomani-Virmouni S, Ramchunder Z, Kendal E, Xiao J. Deciphering the missing links between Friedreich ataxia and multiple sclerosis...

Advancing a novel ASO therapy for Friedreich ataxia cardiomyopathy using a pre-clinical human vascularised cardiac organoid model

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Jarmon G. Lees, Li Li, Haoxiang Zhang, Anne M. Kong, Andrew Treller, Geraldine M. Mitchell, Mirella Dottori, Alice Pebay, Stephen Wilcox, Ma...

Fronto-Cerebellar Connectivity Disruptions and Functional Reorganization in Friedreich’s Ataxia: A Structural and Resting-State fMRI Study

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Ravi Dadsena, Sandro Romanzetti, Stella Andrea Lischewski, Yingua Jing, Dagmar Timmann, Jennifer Faber, Jörg B. Schulz, Kathrin Reetz, Imis ...

Advanced Heart Failure in Friedreich's Ataxia: A Story of Challenges, Opportunities, and Hope

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Advanced Heart Failure in Friedreich's Ataxia: A Story of Challenges, Opportunities, and Hope. Satoshi Miyashita, Anthony Zaki, Kaitlin ...
Wednesday, March 25, 2026

Peripheral frataxin levels govern long-term clinical progression in Friedreich ataxia

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Rummey C, Blair IA, Mesaros C, Rojsajjakul T, Dong Y, Wilmot G, et al. Peripheral frataxin levels govern long-term clinical progression in F...
Saturday, March 21, 2026

Friedreich ataxia transcriptomic dysregulation and identification of cell type-specific biomarkers: A systematic review and meta-analysis

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Friedreich ataxia transcriptomic dysregulation and identification of cell type-specific biomarkers: A systematic review and meta-analysis Ma...

The multifaceted nature of Friedreich ataxia: strategies for comprehensive patient care.

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Raza, M. L., Rawalia, M. A., Fatima, Z., & Zehra, H. (2026). The multifaceted nature of Friedreich ataxia: strategies for comprehensive ...
Monday, March 16, 2026

Design Therapeutics Maps H2 Data Readouts for RESTORE-FA

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MarketBeat. Sun, March 15, 2026. RESTORE-FA targets a demonstrable increase in endogenous frataxin (measured in mRNA and protein, including ...
Thursday, March 12, 2026

Biallelic Truncating DNAH14 Variant in Siblings with Neurodevelopmental Disorder and Predominant Ataxia: Clinical Report and Literature Review

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Baris S, Dogan M, Terali K, Gezdirici A, Eroz R, Yucel PP, Kilic H, Yavas C, Yildirim G, Baris I. Biallelic Truncating DNAH14 Variant in Sib...