Abstract
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.Electronic supplementary materialThe online version of this article (doi:10.1186/s12014-016-9109-x) contains supplementary material, which is available to authorized users.
Highlights
Duchenne muscular dystrophy (DMD) is a severe form of myopathy that affects 1 in 5000–20,000 male births worldwide [1, 2]
Overall using a combination of mass spectrometry based proteomics and SomaScan aptamer array we identified 59 protein biomarkers associated with dystrophin deficiency in serum of DMD patients
Development of “omic” technologies has enabled discovery of novel biomarkers associated with DMD disease progression and response to therapies
Summary
Duchenne muscular dystrophy (DMD) is a severe form of myopathy that affects 1 in 5000–20,000 male births worldwide [1, 2]. A series of studies conducted in the mdx mouse model and DMD patients demonstrated the utility of circulating miRNA as biomarkers to monitor disease severity and response to treatments aiming to restore the missing dystrophin protein [32,33,34].
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