N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy.

ABSTRACT:

Background

Multiple clinical trials to assess the efficacy of AAV-directed gene transfer in participants with Duchenne muscular dystrophy (DMD) are ongoing. The success of these trials currently relies on standard functional outcome measures that may exhibit variability within and between participants, rendering their use as sole measures of drug efficacy challenging. Given this, supportive objective biomarkers may be useful in enhancing observed clinical results. Creatine kinase (CK) is traditionally used as a diagnostic biomarker of DMD, but its potential as a robust pharmacodynamic (PD) biomarker is difficult due to the wide variability seen within the same participant over time. Thus, there is a need for the discovery and validation of novel PD biomarkers to further support and

SUBMITTER: Boehler JF 

PROVIDER: S-EPMC10790446 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature