Investigator: Yi-Wen Chen DMV PhD
Category: Research - Clinical
Biomarkers are biological changes that can be used as indicators of physiological and disease states. Biomarkers can be valuable when the primary cause of the disease is difficult to be examined due to technical or ethical considerations. The goal of this study is to identify protein biomarkers in blood samples from patients with early onset FSHD, which can be used to evaluate treatment efficacy in clinical trials. In this study, we propose to identify biomarkers using blood samples. Three different approaches will be used to identify candidate biomarkers, followed by validation using blood samples from additional patient visits as well as FSHD mouse models. The success of the study will identify biomarkers (blood molecular changes) via a less invasive procedure (blood v.s. muscle biopsy) for evaluating therapeutic efficacy in future clinical studies.