期刊
SCIENTIFIC REPORTS
卷 7, 期 -, 页码 -出版社
NATURE RESEARCH
DOI: 10.1038/s41598-017-17982-y
关键词
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资金
- AFM [17724]
- EU [241665, 305121, 305444]
- ProNova VINN Excellence Centre for Protein Technology (VINNOVA, Swedish Governmental Agency for Innovation Systems)
- Knut and Alice Wallenberg Foundation
- Medical Research Council (MRC) Centre for Neuromuscular Diseases Biobanks (Newcastle) part of EuroBioBank
- Medical Research Council (MRC) Centre for Neuromuscular Diseases Biobanks (London) are part of EuroBioBank
- National Institute for Health Research [NF-SI-0515-10022] Funding Source: researchfish
Duchenne Muscular Dystrophy (DMD) is a severe muscle disorder caused by lack of dystrophin. Predictive biomarkers able to anticipate response to the therapeutic treatments aiming at dystrophin re-expression are lacking. The objective of this study is to investigate Matrix Metalloproteinase-9 (MMP-9) as predictive biomarker for Duchenne. Two natural history cohorts were studied including 168 longitudinal samples belonging to 66 patients. We further studied 1536 samples obtained from 3 independent clinical trials with drisapersen, an antisense oligonucleotide targeting exon 51: an open label study including 12 patients; a phase 3 randomized, double blind, placebo controlled study involving 186 patients; an open label extension study performed after the phase 3. Analysis of natural history cohorts showed elevated MMP-9 levels in patients and a significant increase over time in longitudinal samples. MMP-9 decreased in parallel to clinical stabilization in the 12 patients involved in the open label study. The phase 3 study and subsequent extension study clarified that the decrease in MMP-9 levels was not predictive of treatment response. These data do not support the inclusion of serum MMP-9 as predictive biomarker for DMD patients.
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