Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy.

ABSTRACT:

Background

Duchenne Muscular Dystrophy is a severe, incurable disorder caused by mutations in the dystrophin gene. The disease is characterized by decreased muscle function, impaired muscle regeneration and increased inflammation. In a clinical context, muscle deterioration, is evaluated using physical tests and analysis of muscle biopsies, which fail to accurately monitor the disease progression.

Objectives

This study aims to confirm and asses the value of blood protein biomarkers as disease progression markers using one of the largest longitudinal collection of samples.

Methods

A total of 560 samples, both serum and plasma, collected at three clinical sites are analyzed using a suspension bead array platform to assess 118 proteins targeted by 250 antibodies in micro

SUBMITTER: Strandberg K 

PROVIDER: S-EPMC7369103 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature