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Evaluation of serum...
Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne
- Article/chapterEnglish2017
Publisher, publication year, extent ...
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2017-12-20
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NATURE PUBLISHING GROUP,2017
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printrdacarrier
Numbers
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LIBRIS-ID:oai:DiVA.org:kth-220826
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https://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-220826URI
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https://doi.org/10.1038/s41598-017-17982-yDOI
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https://gup.ub.gu.se/publication/262563URI
Supplementary language notes
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Language:English
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Summary in:English
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Subject category:ref swepub-contenttype
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Subject category:art swepub-publicationtype
Notes
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QC 20180111
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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 and genre
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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, BurcuKTH,Proteomik och nanobioteknologi(Swepub:kth)u1kfjubd
(author)
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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, CristinaKTH,Proteomik och nanobioteknologi(Swepub:kth)u1c02mh9
(author)
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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,1953Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xtulim
(author)
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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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KTHProteomik och nanobioteknologi
(creator_code:org_t)
Related titles
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In:Scientific Reports: NATURE PUBLISHING GROUP72045-2322
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- 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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- 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
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Scientific Repor ...
- By the university
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Royal Institute of Technology
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University of Gothenburg