A tractable vertebrate model that recapitulates key aspects of Friedreich Ataxia
Identification of concordant electrophysiological biomarkers in Friedreich Ataxia
Advancing novel mitochondrial therapies for FA cardiomyopathy: a pragmatic collaboration to move new therapies forward
Measuring ataxia in children with Friedreich ataxia
Evaluation of ARMMs-mediated delivery of Cas9 protein complexed with gRNAs as a non-viral disease-modifying strategy for Friedreich’s Ataxia
Illuminating how SynGRs liberate gene expression from heterochromatin
Detection and enhancement of tissue NAD+ levels in Friedreich’s Ataxia
A New Ultrasensitive Single-Molecule Assay for Frataxin Measurement
Rescue of Friedreich’s Ataxia cells by DNA methylation editing of the FXN gene
Understanding ketogenesis in FRDA: Pathophysiology, biomarkers and nutritional therapies
Investigating epigenetic silencing in Friedreich’s Ataxia
Therapeutic activity of a haematopoietic stem cell delivered tissue penetrating peptide in a Friedreich’s ataxia mouse model