Sökning: onr:"swepub:oai:DiVA.org:kth-220826" > Evaluation of serum...
Fältnamn | Indikatorer | Metadata |
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000 | 04103naa a2200673 4500 | |
001 | oai:DiVA.org:kth-220826 | |
003 | SwePub | |
008 | 180111s2017 | |||||||||||000 ||eng| | |
009 | oai:gup.ub.gu.se/262563 | |
024 | 7 | a https://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-2208262 URI |
024 | 7 | a https://doi.org/10.1038/s41598-017-17982-y2 DOI |
024 | 7 | a https://gup.ub.gu.se/publication/2625632 URI |
040 | a (SwePub)kthd (SwePub)gu | |
041 | a engb eng | |
042 | 9 SwePub | |
072 | 7 | a ref2 swepub-contenttype |
072 | 7 | a art2 swepub-publicationtype |
100 | 1 | a Lourbakos, A.4 aut |
245 | 1 0 | a Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne |
264 | c 2017-12-20 | |
264 | 1 | b NATURE PUBLISHING GROUP,c 2017 |
338 | a print2 rdacarrier | |
500 | a QC 20180111 | |
520 | a 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. | |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Klinisk laboratoriemedicin0 (SwePub)302232 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Clinical Laboratory Medicine0 (SwePub)302232 hsv//eng |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Pediatrik0 (SwePub)302212 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Pediatrics0 (SwePub)302212 hsv//eng |
700 | 1 | a Yau, N.4 aut |
700 | 1 | a de Bruijn, P.4 aut |
700 | 1 | a Hiller, M.4 aut |
700 | 1 | a Kozaczynska, K.4 aut |
700 | 1 | a Jean-Baptiste, R.4 aut |
700 | 1 | a Reza, M.4 aut |
700 | 1 | a Wolterbeek, R.4 aut |
700 | 1 | a Koeks, Z.4 aut |
700 | 1 | a Ayoglu, Burcuu KTH,Proteomik och nanobioteknologi4 aut0 (Swepub:kth)u1kfjubd |
700 | 1 | a de Klerk, D.4 aut |
700 | 1 | a Campion, G.4 aut |
700 | 1 | a Zaharieva, I.4 aut |
700 | 1 | a Nadarajah, V. D.4 aut |
700 | 1 | a Nilsson, P.4 aut |
700 | 1 | a Al-Khalili Szigyarto, Cristinau KTH,Proteomik och nanobioteknologi4 aut0 (Swepub:kth)u1c02mh9 |
700 | 1 | a Muntoni, F.4 aut |
700 | 1 | a Lochmuller, H.4 aut |
700 | 1 | a Verschuuren, J. J.4 aut |
700 | 1 | a Goemans, N.4 aut |
700 | 1 | a Tulinius, Mar,d 1953u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xtulim |
700 | 1 | a Niks, E. H.4 aut |
700 | 1 | a de Kimpe, S.4 aut |
700 | 1 | a Aartsma-Rus, A.4 aut |
700 | 1 | a 't Hoen, Peter A. C.4 aut |
700 | 1 | a Spitali, P.4 aut |
710 | 2 | a KTHb Proteomik och nanobioteknologi4 org |
773 | 0 | t Scientific Reportsd : NATURE PUBLISHING GROUPg 7q 7x 2045-2322 |
856 | 4 | u https://www.nature.com/articles/s41598-017-17982-y.pdf |
856 | 4 8 | u https://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-220826 |
856 | 4 8 | u https://doi.org/10.1038/s41598-017-17982-y |
856 | 4 8 | u https://gup.ub.gu.se/publication/262563 |
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