A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocol.

ABSTRACT:

Introduction

Drug development for neurodegenerative diseases such as Friedreich's ataxia (FRDA) is limited by a lack of validated, sensitive biomarkers of pharmacodynamic response in affected tissue and disease progression. Studies employing neuroimaging measures to track FRDA have thus far been limited by their small sample sizes and limited follow up. TRACK-FA, a longitudinal, multi-site, and multi-modal neuroimaging natural history study, aims to address these shortcomings by enabling better understanding of underlying pathology and identifying sensitive, clinical trial ready, neuroimaging biomarkers for FRDA.

Methods

200 individuals with FRDA and 104 control participants will be recruited across seven international study sites. Inclusion criteria for participants with ge

SUBMITTER: Georgiou-Karistianis N 

PROVIDER: S-EPMC9678384 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature