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Evaluation of serum...
Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne
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Lourbakos, A. (author)
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Yau, N. (author)
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de Bruijn, P. (author)
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Hiller, M. (author)
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Kozaczynska, K. (author)
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Jean-Baptiste, R. (author)
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Reza, M. (author)
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Wolterbeek, R. (author)
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Koeks, Z. (author)
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- Ayoglu, Burcu (author)
- KTH,Proteomik och nanobioteknologi
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de Klerk, D. (author)
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Campion, G. (author)
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Zaharieva, I. (author)
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Nadarajah, V. D. (author)
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Nilsson, P. (author)
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- Al-Khalili Szigyarto, Cristina (author)
- KTH,Proteomik och nanobioteknologi
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Muntoni, F. (author)
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Lochmuller, H. (author)
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Verschuuren, J. J. (author)
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Goemans, N. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Niks, E. H. (author)
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de Kimpe, S. (author)
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Aartsma-Rus, A. (author)
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't Hoen, Peter A. C. (author)
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Spitali, P. (author)
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(creator_code:org_t)
- 2017-12-20
- 2017
- English.
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In: Scientific Reports. - : NATURE PUBLISHING GROUP. - 2045-2322. ; 7
- Related links:
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https://www.nature.c...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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- 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 headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Klinisk laboratoriemedicin (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Clinical Laboratory Medicine (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Publication and Content Type
- ref (subject category)
- art (subject category)
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To the university's database
- By the author/editor
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Lourbakos, A.
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Yau, N.
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de Bruijn, P.
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Hiller, M.
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Kozaczynska, K.
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Jean-Baptiste, R ...
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show more...
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Reza, M.
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Wolterbeek, R.
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Koeks, Z.
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Ayoglu, Burcu
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de Klerk, D.
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Campion, G.
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Zaharieva, I.
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Nadarajah, V. D.
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Nilsson, P.
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Al-Khalili Szigy ...
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Muntoni, F.
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Lochmuller, H.
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Verschuuren, J. ...
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Goemans, N.
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Tulinius, Mar, 1 ...
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Niks, E. H.
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de Kimpe, S.
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Aartsma-Rus, A.
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't Hoen, Peter A ...
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Spitali, P.
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Clinical Laborat ...
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
- Articles in the publication
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Scientific Repor ...
- By the university
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Royal Institute of Technology
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University of Gothenburg