Data-derived wearable digital biomarkers predict Frataxin gene expression levels and longitudinal disease progression in Friedreich’s Ataxia

Aldo Faisal, Balasundaram Kadirvelu, Constantinos Gavriel, Sathiji Nageshwaran, Ping Kei Jackson Chan, Stavros Athanasopoulos, Paola Giunti, Valeria Ricotti, Thomas Voit, Richard Festenstein; Research Square; 2021. DOI: 10.21203/rs.3.rs-737100/v1. 

Our wearable digital biomarker can accurately predict for each patient their personal FXN gene expression levels, demonstrating the sensitivity of our approach and the importance of FXN levels in FA. Therefore, our data-derived biomarker approach can not only cross-sectionally predict disease and their gene expression levels but also their longitudinal disease trajectory: it is sensitive and accurate enough to detect disease progression with much fewer subjects or shorter time scales than existing primary endpoints. Our work demonstrates that data-derived wearable biomarkers have the potential to substantially reduce clinical trial durations and a first in-human demonstration of reconstructing FXN gene expression levels from behavioral data alone.