Anodal cerebellar tDCS does not alter beta oscillations or corticokinematic coherence in Friedreich’s ataxia and healthy participants

Christian Georgiev, Mathieu Bourguignon, Scott J. Mongold, Lousin Moumdjian, Pierre Cabaraux, Gilles Naeije, Anodal cerebellar tDCS does not alter beta oscillations or corticokinematic coherence in Friedreich’s ataxia and healthy participants, Clinical Neurophysiology, Volume 190, 2026, 2111961, ISSN 1388-2457, doi:10.1016/j.clinph.2026.2111961. 

Anodal ctDCS improved FA motor symptom severity without altering SM1 excitability. 

Anodal ctDCS has a therapeutic effect in FA, however, the neurophysiology of this effect is complex and requires further investigation.

New Progress Toward Public Reimbursement of SKYCLARYS™ for People Living with Friedreich Ataxia in Quebec

TORONTO, June 8, 2026 /CNW/ - Biogen Canada Inc. is pleased to announce a positive outcome from the Institut national d'excellence en santé et en services sociaux (INESSS) re-evaluation of SKYCLARYS™ (omaveloxolone), recognizing the therapeutic value of the treatment and establishing reimbursement criteria for eligible patients living with Friedreich ataxia (FA) in Quebec. This outcome represents a positive step toward public reimbursement in the province for the only approved treatment in Canada for Friedreich ataxia.

MRI end-points for clinical trials in ataxias: recommendations from the Ataxia Global Initiative MRI Biomarkers Working Group

Öz, G., Cocozza, S., Rezende, T.J.R. et al. MRI end-points for clinical trials in ataxias: recommendations from the Ataxia Global Initiative MRI Biomarkers Working Group. Nat Rev Neurol (2026). doi:10.1038/s41582-026-01218-7 

In this Consensus Statement, the Ataxia Global Initiative MRI Biomarkers Working Group critically reviews candidate MRI end-points for trials in the most common spinocerebellar ataxias (SCA1, SCA2 and SCA3) and Friedreich ataxia and provides evidence-based, disease-specific recommendations for the selection of MRI end-points for trials in these diseases.

New clinical trial for vatiquinone

May 29, 2026 PTC Therapeutics. 

Dear Friedreich's Ataxia Community - PTC Therapeutics We are excited to share that we will be initiating a new clinical trial for vatiquinone for the treatment of individuals living with Friedreich's ...

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

Yameogo P, Gerhart BJ, Sentmanat MF, Neilson A, Cui X, Verma M, Lynch DR, Napierala JS, Napierala M. Generation of Friedreich's ataxia induced pluripotent stem cells carrying the FXN c.165 + 5G>C splicing mutation. Stem Cell Res. 2026 Jun;93:103966. doi: 10.1016/j.scr.2026.103966. Epub 2026 Mar 16. PMID: 41865460.

We generated induced pluripotent stem cells from blood lymphocytes from a FRDA patient carrying the FXN c.165 + 5G > C point mutation, which interferes with canonical splicing of intron 1 of the FXN gene. These cells allow for development of therapeutic approaches that target splicing defect in FRDA.

Xavier disease (Friedreich's ataxia variant) - Symptoms, Causes, Treatment & Prevention

📅 Updated: April 2026. Xavier disease is a very rare, autosomal‑recessive neuro‑degenerative disorder that shares many clinical features with classic Friedreich’s ataxia (FA) but is caused by distinct genetic changes. The name “Xavier disease” originates from the first family in which the condition was identified (the Xavier family) and is sometimes listed in literature as FA‑variant or Friedreich‑type ataxia with atypical genotype.

Targeting competitive Fe–S regulation to treat Friedreich’s ataxia

Campos J, Ferreira J. Targeting competitive Fe–S regulation to treat Friedreich’s ataxia. Trends in Pharmacological Sciences, 2026; 0 DOI: 10.1016/j.tips.2026.04.010

Recent discoveries reveal that frataxin (FXN) and ferredoxin 2 (FDX2) competitively regulate mitochondrial iron–sulfur (Fe–S) cluster biosynthesis through their binding to the cysteine desulfurase NFS1 and the iron-sulfur cluster scaffold protein ISCU2 complex. Here, we discuss the potential of rationally designed peptide inhibitors targeting the FDX2–NFS1 interaction as a strategy to mitigate FXN deficiency and restore Fe–S cluster biosynthesis.

A phosphorylated variant of the mast/stem cell growth factor receptor KIT is upregulated in dorsal root ganglia of Friedreich ataxia

Koeppen AH, Mazurkiewicz JE, Feustel PJ, Pelech S, Sutter C, Fu Q, Lin Q. A phosphorylated variant of the mast/stem cell growth factor receptor KIT is upregulated in dorsal root ganglia of Friedreich ataxia. Histol Histopathol. 2026 May 20:25093. doi: 10.14670/HH-25-093. Epub ahead of print. PMID: 42158966. 

Friedreich ataxia (FA) causes hypoplasia of nerve cells in dorsal root ganglia (DRG). Beyond hypoplasia, however, the lesion in DRG includes disorganization and proliferation of satellite cells, formation of residual nodules, and neuronophagia. 

KIT is a proto-oncogenic protein with prominent roles in hematopoiesis including mast cell proliferation. In conclusion, proteomic analysis confirms the prominent participation of a new truncated KIT in satellite cells in the pathogenesis of FA in DRG.

Psychological resilience in patients with Friedreich ataxia: a 6-year longitudinal analysis

Ruth Eumann, Janna Krahe, Imis Dogan, Ana Sofia Costa, Jörg B Schulz, Stella A Lischewski, Kathrin Reetz; FACROSS Study Group. Psychological resilience in patients with Friedreich ataxia: a 6-year longitudinal analysis. Neurol Res Pract. 2026 May 19;8(1):40. doi: 10.1186/s42466-026-00499-z. PubMed ID: 42157313 

 Individuals with Friedreich ataxia display reduced resilience compared to healthy controls, which is associated with greater levels of depression and anxiety. Resilience remained stable over the disease course but appeared to decline during the COVID-19 pandemic, underlining the importance of strengthening resilience and mental well-being in this population.

Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich’s ataxia models

Pido-Lopez J, Moula S, Shaban E ... Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich’s ataxia models Cell Reports Medicine, 2026; 0 doi:10.1016/j.xcrm.2026.102803