Assessments of disease progression and response to therapies in Duchenne muscular dystrophy (DMD) patients remain challenging. Current DMD patient assessments include complex physical tests and invasive procedures such as muscle biopsies, which are not suitable for young children. Defining alternative, less invasive and objective outcome measures to assess disease progression and response to therapy will aid drug development and clinical trials in DMD. In this review we highlight advances in development of non-invasive blood circulating biomarkers as a means to assess disease progression and response to therapies in DMD.
Keywords: Biomarkers; Clinical outcomes; Duchenne muscular dystrophy; Mass spectrometry; Pharmacodynamic biomarkers; Proteins; SomaScan; Surrogate biomarkers; miRNA.