Skeletal Muscle

Skeletal Muscle is excited to feature the recently published paper: "N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy" by Solid Biosciences. This work investigates the use of non-invasive markers to assess the efficacy of microdystrophin gene therapy and to supplement traditional outcomes measures. The N-terminal fragment of titin is elevated in DMD biofluids but was reduced in conjunction with microdystrophin expression in the skeletal muscle and localization to the sarcolemma. Further, this reduction in N-terminal titin was sensitive to lower levels of expressed microdystrophin in the skeletal muscle when compared to serum CK. Biomarkers from non-invasive biofluids, like the N-terminal fragment of titin, have the potential to provide additional confidence in drug efficacy, to help drug developers reach decisions faster, and to reduce the burden of clinical trial participation for patients and their families. Image Created with Biorender.