News & Blog

Application of RSwitch technology to improve Friedreich’s ataxia viral gene therapy safety and efficacy: control of FXN transgene expression through a small molecule regulation

Development of Epigenetic Editors for Frataxin Reactivation

Quantifying sitting balance in children and adults with Friedreich ataxia – development of clinical and instrumented measures

Design and synthesis of SynGR library to probe into the role of NSD-2,3 proteins in FXN expression in FRDA

Impact of cerebellar transcranial direct current stimulation on FA brain and cerebellar neurophysiological fingerprints and on clinical symptoms assessed by multimodal evaluations

Understanding the Pathogenesis of Friedreich’s Ataxia by Single-Cell Integration of Gene Expression, Epigenetics and Connectivity in the Cerebellar Nuclei

Enhancing Gene Therapy Delivery to the Brain via Human Blood-Brain Barrier Receptors

Mitochondrial Enzyme Activity and Biomarker Evaluation in FA Treatment (MEABET-FA)

Shortening GAA repeats in Friedreich’s Ataxia

NRF2 and MAO-Ai therapy to improve cardiac health and prevent arrhythmias in Friedreich’s ataxia

Postnatal development of the cerebellar circuitry in the KIKO mouse model of Friedreich’s ataxia

Developing the novel investigational mitochondrial therapeutic SBT-589 for Friedreich’s ataxia