Defining and targeting cardiac lipid dysregulation in Friedreich ataxia using human iPSC-derived cardiac models
CBM-01, A Novel Inhibitor of Lipid Peroxidation for Targeting Iron-mediated Pathology in FA Cardiomyopathy
AIMs_Play: Predicting Disease Progression in Pediatric Friedreich Ataxia with Generative AI-Enabled Platform
Neuro-otology: Diffusion MRI and audiological biomarkers for Friedreich ataxia disease progression
RNA Writing Trans-Splicing for Enhancing Frataxin Translation
Characterization of Fatigue in Patients with Friedreich’s Ataxia by Assessment of Mitochondrial Function in Skeletal and Cardiac Muscle
Correcting Cell-Autonomous Neuroinflammatory Phenotypes in Friedreich’s Ataxia Microglia Using iPSC-Based Approaches
Development and Validation of a New Friedreich’s Ataxia Isogenic Control Mouse
Structural Dynamics of the Iron-Sulfur Cluster Assembly Machinery in Patient-Derived FRDA Cells Using Super-Resolution Microscopy
Investigating satellite glial cell phenotypes and functions in the pathogenesis of Friedreich’s ataxia
Discovery of genetic suppressor mutations that rescue frataxin deficiency
Pre-clinical assessment of protein replacement therapy in the central nervous system of Friedreich’s ataxia mouse models