Pharmacological treatments for Friedreich ataxia ( Cochrane Database of Systematic Reviews 2026)

Lyons S, Kearney M, Fahey MC, Janjal P, Pandolfo M, Patton P. Pharmacological treatments for Friedreich ataxia. Cochrane Database of Systematic Reviews 2026, Issue 5. Art. No.: CD007791. DOI: 10.1002/14651858.CD007791.pub5. Accessed 12 May 2026.

In this updated Cochrane systematic review, meta‐analysis of results on the ataxia rating scale showed that pharmacological treatments probably make little or no difference compared with placebo after 12 months of treatment. Given this result, the probable improvement that we found in upper limb dexterity was unexpected. Treatment‐emergent adverse events leading to cessation of medication or death may be no more common in treatment groups than placebo groups as there were few adverse events detected in the treated groups. However, the studies may not have detected all rare and serious adverse events.

Transcription and Cohesin Direct Domain Boundary Spatial Positioning and are Linked to Friedreich's Ataxia

Ashley Karnay, Ricardo Linares-Saldana, Qiaohong Wang, Zachary Gardner, Jialiu A. Liang, Garrett T. Santini, Krishna Kumar Haridhasapavalan, Son C. Nguyen, Siewert Hugelier, Bhavana Shewale, Masato T. Kanemaki, Jill S. Napierala, Marek Napierala, Robert B. Wilson, Nicole Dubois, Andrey Poleshko, Wonho Kim, Parisha P. Shah, Melike Lakadamyali, Eric F. Joyce, Rajan Jain, Transcription and cohesin direct domain boundary spatial positioning and are linked to Friedreich’s ataxia, Molecular Cell, 2026, DOI: 10.1016/j.molcel.2026.04.019. 

These results suggest that gene silencing in Friedreich's ataxia is reinforced by where the gene sits in the nucleus," said Ashley Karnay, Ph.D., a postdoctoral fellow in Cardiovascular Medicine and Cell & Developmental Biology and the study's lead author. "By changing that positioning, we can partially restore FXN gene activity in diseased cells." 

 While the findings are early and not a treatment, they point to genome organization itself as a contributor to disease and raise the exciting possibility that future therapies could work by changing how the DNA is organized inside the nucleus.