Limiting intestinal iron absorption rescues glial defects and extends lifespan in a Drosophila model of Friedreich ataxia

Limiting intestinal iron absorption rescues glial defects and extends lifespan in a Drosophila model of Friedreich ataxia Ema TURKI, Estelle JULLIAN, Pierre DELAMOTTE, Anne FILIPE, Laura TIXIER CARDOSO, Sandrine MIDDENDORP, Elodie MARTIN, Veronique Monnier bioRxiv 2026.06.04.730074; doi:10.64898/2026.06.04.730074 

 Reducing intestinal iron uptake, either through treatment with bathophenanthroline disulfonic acid (BPS), an extracellular iron chelator, or by gut-specific silencing of the iron transporter Malvolio, nearly doubled fly survival. BPS treatment also improved sensitivity to dietary iron, enhanced locomotor performance, fully restored normal brain size, and prevented glial alterations. Altogether, our findings identify glial cells as early and preferential targets of frataxin deficiency in an iron-dependent manner and support the in vivo relevance of intestinal iron uptake as a potential modulator of disease severity in FRDA.

Chemical modulation of Miro1 alleviates cell-type-specific vulnerabilities in Friedreich’s ataxia

Chandra S, Kwak C, Du Z ... Chemical modulation of Miro1 alleviates cell-type-specific vulnerabilities in Friedreich’s ataxia, Cell Chemical Biology, 2026; 0.  DOI: 10.1016/j.chembiol.2026.05.004

MR3 treatment modulates molecular signatures in a cell-type-dependent manner, altering pathways related to cardiac contractility in cardiomyocytes and synaptic function in sensory neurons. Mechanistically, MR3 reduces mitochondrial reactive oxygen species and restores membrane potential in FA sensory neurons via potential allosteric reshaping of Miro1 protein. We expand the chemical diversity of this scaffold by conducting ligand-based virtual screening of over 3 billion compounds and identifying previously uncharacterized Miro1 ligands with improved docking and neuroprotective capacity.

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.