Tracking disease progression non-invasively in Duchenne and Becker muscular dystrophies.

ABSTRACT:

Background

Analysis of muscle biopsies allowed to characterize the pathophysiological changes of Duchenne and Becker muscular dystrophies (D/BMD) leading to the clinical phenotype. Muscle tissue is often investigated during interventional dose finding studies to show in situ proof of concept and pharmacodynamics effect of the tested drug. Less invasive readouts are needed to objectively monitor patients' health status, muscle quality, and response to treatment. The identification of serum biomarkers correlating with clinical function and able to anticipate functional scales is particularly needed for personalized patient management and to support drug development programs.

Methods

A large-scale proteomic approach was used to identify serum biomarkers describing pathophysiolo

SUBMITTER: Spitali P 

PROVIDER: S-EPMC6104105 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature