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

Kwa FAA, Anjomani-Virmouni S, Ramchunder Z, Kendal E, Xiao J. Deciphering the missing links between Friedreich ataxia and multiple sclerosis for targeted drug development. Drug Discov Today. 2026 Mar 16;31(3):104644. doi: 10.1016/j.drudis.2026.104644. Epub ahead of print. PMID: 41850598. 

 FA and MS appear to share some overlapping molecular mechanisms, including iron and lipid dysregulation, mitochondrial dysfunction, oxidative stress, and neuroinflammation. Recent research, including comparative transcriptomic analyses, offers valuable insights into shared disease pathways, with implications for potential biomarkers and therapeutic targets. In this review, we explore the shared pathological features and disease mechanisms in FA and MS, highlighting how delineating these shared pathways could inform early diagnostic strategies and support the development of targeted, mechanism-based interventions, including opportunities for drug repurposing.

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

Jarmon G. Lees, Li Li, Haoxiang Zhang, Anne M. Kong, Andrew Treller, Geraldine M. Mitchell, Mirella Dottori, Alice Pebay, Stephen Wilcox, Mark Chong, Roger Peverill, Martin Delatyki, Jeffrey M. Pullin, Davis McCarthy, Jill S. Napierala, Marek Napierala, Shiang Y. Lim, Advancing a novel ASO therapy for Friedreich ataxia cardiomyopathy using a pre-clinical human vascularised cardiac organoid model, Journal of Molecular and Cellular Cardiology Plus, Volume 15, Supplement, 2026, 100527, ISSN 2772-9761, doi:10.1016/j.jmccpl.2025.100527. 

ASO-mediated knockdown of MEG3 reversed EC angiogenic dysfunction, limited SMC migration, reversed mitochondrial dysfunction, and prevented cardiac injury in a human vascularized cardiac organoid model. These findings suggest that MEG3 may be a promising novel therapy for treating cardiovascular disease in FA.

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

Ravi Dadsena, Sandro Romanzetti, Stella Andrea Lischewski, Yingua Jing, Dagmar Timmann, Jennifer Faber, Jörg B. Schulz, Kathrin Reetz, Imis Dogan, Fronto-Cerebellar Connectivity Disruptions and Functional Reorganization in Friedreich’s Ataxia: A Structural and Resting-State fMRI Study, NeuroImage, 2026, 121872, ISSN 1053-8119, doi:10.1016/j.neuroimage.2026.121872.

While regional atrophy is known to be associated with symptoms, functional network alterations may represent a critical pathological mechanism; however, their specific contribution to motor and cognitive impairment remains unclear.

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

Advanced Heart Failure in Friedreich's Ataxia: A Story of Challenges, Opportunities, and Hope. Satoshi Miyashita, Anthony Zaki, Kaitlin Schlabach, Gabriel Kim, Juliane K. Vierecke, David Lynch, Carmela Tan, Eileen Hsich, and Paulino Alvarez; JACC: Case Reports, 2026, 107391, ISSN 2666-0849, doi:10.1016/j.jaccas.2026.107391.

Heart failure is the leading cause of death in patients with FRDA. For those with high-risk features for adverse cardiovascular outcomes, careful monitoring of disease progression and early evaluation for advanced heart failure therapies are warranted.