Biomarkers in Duchenne Muscular Dystrophy: Current Status and Future Directions.

ABSTRACT:  Duchenne muscular dystrophy is a severe, X-linked disease characterized by decreased muscle mass and function in children. Genetic and biochemical research over the years has led to the characterization of the cause and the pathophysiology of the disease. Moreover, the elucidation of genetic mechanisms underlining Duchenne muscular dystrophy has allowed for the design of innovative personalized therapies.The identification of specific, accurate, and sensitive biomarkers is becoming crucial for evaluating muscle disease progression and response to therapies, disease monitoring, and the acceleration of drug development and related regulatory processes.This review illustrated the up-to-date progress in the development of candidate biomarkers in DMD at the level of proteins, metabolites, micr

SUBMITTER: Fortunato F 

PROVIDER: S-EPMC10657716 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature