Evidence map›Paper›PMID 29263366›Full record

ArticleScientific reports2017

Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne.

A Lourbakos, N Yau, P de Bruijn, M Hiller, K Kozaczynska, R Jean-Baptiste, M Reza, R Wolterbeek, Z Koeks, B Ayoglu and 16 more

Abstract read
In one paragraph

Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed.

  1. Trial
  2. Article
  3. Article
  4. Serum protein biomarker signature of Duchenne muscular dystrophy.European journal of translational myology · 2025
    Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Review
  11. Review
  12. Circulating Biomarkers in Muscular Dystrophies: Disease and Therapy Monitoring.Molecular therapy. Methods & clinical development · 2020
    Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. Non-Glycanated Biglycan and LTBP4: Leveraging the extracellular matrix for Duchenne Muscular Dystrophy therapeutics.Matrix biology : journal of the International Society for Matrix Biology · 2018
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors.

A LourbakosBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
N YauBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
P de BruijnBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
M HillerDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
K KozaczynskaBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
R Jean-BaptisteBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
M RezaJohn Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
R WolterbeekDepartment of Medical Statistics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
Z KoeksDepartment of Neurology, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
B AyogluDepartment of Proteomics, School of Biotechnology, KTH-Royal Institute of Technology, Stockholm, Sweden.
D de KlerkDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
G CampionBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
I ZaharievaDubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Institute of Child Health, London, UK.
V D NadarajahDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
P NilssonDepartment of Proteomics, School of Biotechnology, KTH-Royal Institute of Technology, Stockholm, Sweden.
C Al-Khalili SzigyartoDepartment of Proteomics, School of Biotechnology, KTH-Royal Institute of Technology, Stockholm, Sweden.
F MuntoniDubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Institute of Child Health, London, UK.
H LochmüllerJohn Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
J J VerschuurenDepartment of Neurology, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.ORCID 0000-0002-4572-1501
N GoemansChild Neurology, University Hospitals Leuven, Leuven, Belgium.
M TuliniusDepartment of Pediatrics, University of Gothenburg, Queen Silvia Children's Hospital, Gothenburg, Sweden.
E H NiksDepartment of Neurology, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
S de KimpeBioMarin Nederland BV, J.H. Oortweg 21, 2333 CH, Leiden, The Netherlands.
A Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.
Peter A C 't HoenDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands.ORCID 0000-0003-4450-3112
P SpitaliDepartment of Human Genetics, Leiden University Medical Center, PO Box 9600, 2300 RC, Leiden, The Netherlands. p.spitali@lumc.nl.ORCID 0000-0003-2783-688X

Funding

Medical Research Council
6 · The paper itself

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.

Indexed as

AdolescentAdultBiomarkersChildChild, PreschoolClinical Trials, Phase III as TopicDouble-Blind MethodDystrophinExonsFemaleHumansLongitudinal StudiesMaleMatrix Metalloproteinase 9Muscular Dystrophy, DuchenneOligonucleotides, AntisenseBiomarkersDystrophinMatrix Metalloproteinase 9Oligonucleotides, Antisense

Identifiers

PMID29263366
PMCPMC5738430

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.