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Sci Rep ; 7(1): 17888, 2017 12 20.
Article in English | MEDLINE | ID: mdl-29263366

ABSTRACT

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.


Subject(s)
Biomarkers/blood , Matrix Metalloproteinase 9/blood , Muscular Dystrophy, Duchenne/blood , Muscular Dystrophy, Duchenne/genetics , Oligonucleotides, Antisense/genetics , Adolescent , Adult , Child , Child, Preschool , Clinical Trials, Phase III as Topic , Double-Blind Method , Dystrophin/genetics , Exons/genetics , Female , Humans , Longitudinal Studies , Male , Randomized Controlled Trials as Topic , Young Adult
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