4.3 Review

Clinical utility of serum biomarkers in Duchenne muscular dystrophy

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

Funding

  1. National Institutes of Health [R01AR062380, P50AR060836, P30HD040677, U54HD071601]
  2. DoD Grant [W81XWH-15-1-0265]
  3. AFM-Telethon
  4. Muscular Dystrophy Association
  5. Foundation to Eradicate Duchenne
  6. Clark Charitable Foundation

Ask authors/readers for more resources

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.

Authors

I am an author on this paper
Click your name to claim this paper and add it to your profile.

Reviews

Primary Rating

4.3
Not enough ratings

Secondary Ratings

Novelty
-
Significance
-
Scientific rigor
-
Rate this paper

Recommended

No Data Available
No Data Available