Journal
CLINICAL PROTEOMICS
Volume 13, Issue -, Pages -Publisher
BMC
DOI: 10.1186/s12014-016-9109-x
Keywords
Duchenne muscular dystrophy; Biomarkers; miRNA; Proteins; Pharmacodynamic biomarkers; Surrogate biomarkers; Clinical outcomes; Mass spectrometry; SomaScan
Categories
Funding
- National Institutes of Health [R01AR062380, P50AR060836, P30HD040677, U54HD071601]
- DoD Grant [W81XWH-15-1-0265]
- AFM-Telethon
- Muscular Dystrophy Association
- Foundation to Eradicate Duchenne
- Clark Charitable Foundation
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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.
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