Evaluation of a serum protein signature as monitoring biomarker for Duchenne Muscular Dystrophy in a long-term clinical trial with corticosteroids.

ABSTRACT:

Objective

Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder for which monitoring biomarkers are urgently needed. We aimed to evaluate whether proteins in serum can accurately monitor patients' function within the duration of a clinical trial.

Methods

In this study, we evaluated longitudinal serum proteins of DMD patients participating to the FOR-DMD clinical trial, comparing daily and intermittent corticosteroid regimens in boys aged 4-8 years at baseline. Using the aptamer-based protein platform SomaScan, we profiled 1500 proteins. Associations between protein levels and motor function outcomes, such as Rise from the Floor Velocity (RFV), 10-Meter Run/Walk Velocity (10MRWV), and North Star Ambulatory Assessment (NSAA), were assessed using linear mixe

SUBMITTER: Degan C 

PROVIDER: S-EPMC12772657 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature