Zobrazeno 1 - 10
of 146
pro vyhledávání: '"Lourbakos A"'
Autor:
Nathalie M Goemans, Már Tulinius, Marleen van den Hauwe, Anna-Karin Kroksmark, Gunnar Buyse, Rosamund J Wilson, Judith C van Deutekom, Sjef J de Kimpe, Afrodite Lourbakos, Giles Campion
Publikováno v:
PLoS ONE, Vol 11, Iss 9, p e0161955 (2016)
BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.MethodsThis 188-week open-label extension
Externí odkaz:
https://doaj.org/article/3e55aff068e14473843a4a30a61a1672
Akademický článek
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Autor:
Chantal Beekman, Jessica A Sipkens, Janwillem Testerink, Stavros Giannakopoulos, Dyonne Kreuger, Judith C van Deutekom, Giles V Campion, Sjef J de Kimpe, Afrodite Lourbakos
Publikováno v:
PLoS ONE, Vol 9, Iss 9, p e107494 (2014)
Duchenne muscular dystrophy (DMD) is characterized by the absence or reduced levels of dystrophin expression on the inner surface of the sarcolemmal membrane of muscle fibers. Clinical development of therapeutic approaches aiming to increase dystroph
Externí odkaz:
https://doaj.org/article/cd13d08f1d8d4544abd4ec444d29d804
Autor:
Zhengning Lin, G. Campion, Sjef J. de Kimpe, Afrodite Lourbakos, Kevin M. Flanigan, Craig M. McDonald, Brenda Wong, R. Wilson
Publikováno v:
Annals of Clinical and Translational Neurology
Objective This double‐blind, randomized, placebo‐controlled Phase 2 study (NCT01462292) assessed the 24‐week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24‐week off‐dose pers
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b33fe897de2eb0f77970f6397f5a42d6
https://doi.org/10.1002/acn3.579
https://doi.org/10.1002/acn3.579
Publikováno v:
In Polymer 2004 45(10):3305-3312
Autor:
Lourbakos, Afrodite, Yuan, YuPing, Jenkins, Alison L., Travis, James, Andrade-Gordon, Patricia, Santulli, Rosemary, Potempa, Jan, Pike, Robert N. *
Publikováno v:
In Blood 15 June 2001 97(12):3790-3797
Autor:
S. de Kimpe, D. de Klerk, Monika Hiller, Jan J.G.M. Verschuuren, Annemieke Aartsma-Rus, R. Jean-Baptiste, Francesco Muntoni, Mar Tulinius, Afrodite Lourbakos, Peter Nilsson, Zaïda Koeks, K. Kozaczynska, G. Campion, Nathalie Goemans, Pietro Spitali, Burcu Ayoglu, Ron Wolterbeek, Vishna Devi Nadarajah, N. Yau, Peter A C 't Hoen, Erik H. Niks, Mojgan Reza, Hanns Lochmüller, P. de Bruijn, C. Al-Khalili Szigyarto, Irina Zaharieva
Publikováno v:
Scientific Reports
Scientific Reports, 7
Scientific Reports, Vol 7, Iss 1, Pp 1-10 (2017)
Scientific Reports, 7
Scientific Reports, Vol 7, Iss 1, Pp 1-10 (2017)
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0e00690df27119c632541d5bb3e76d3a
https://hdl.handle.net/1887/115152
https://hdl.handle.net/1887/115152
Autor:
Nathalie Goemans, Eugenio Mercuri, Elena Belousova, Hirofumi Komaki, Alberto Dubrovsky, Craig M. McDonald, John E. Kraus, Afrodite Lourbakos, Zhengning Lin, Giles Campion, Susanne X. Wang, Craig Campbell, A. Araujo, E. Bertini, P. Born, C. Cances, B. Chabrol, J.-H. Chae, J. Colomer Oferil, G.P. Comi, J.-M. Cuisset, G. D'Anjou, I. Desguerre, R. Erazo Torricelli, R. Escobar, D. Feder, A. Ferlini, R. Giugliani, E. Henricson, A. Herczegfalvi, Y.-J. Jong, S. Kimura, J.-B. Kirschner, K. Kleinsteuber, A. Kostera-Pruszczyk, M. Kudr, W. Mueller-Felber, E.H. Niks, K. Ogata, C. Palermo, M. Pane, I. Pascual, Y. Pereon, S. Raskin, M. Rasmussen, U. Reed, U. Schara, K. Selby, C. Sobreira, Y. Takeshima, J.J. Vilchez Padilla, G. Vita, P. Vondracek, G. Wiegand, E. Wilichowski
Publikováno v:
Paediatrics Publications
Neuromuscular Disorders
Neuromuscular Disorders, 28(1), 4-15
Neuromuscular Disorders
Neuromuscular Disorders, 28(1), 4-15
This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated efficacy and safety of subcutaneous drisapersen 6 mg/kg/week in 186 ambulant boys aged >=5 years, with Duchenne muscular dystrophy (DMD) resulting from an e
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2b23f19cae216fd0ab3f5c58c553a0a7
https://pubmed.ncbi.nlm.nih.gov/29203355
https://pubmed.ncbi.nlm.nih.gov/29203355
Autor:
Nicolas Deconinck, Steve S Hood, Haluk Topaloglu, Lia Liefaard, C. Wardell, Rosamund Wilson, Sjef J. de Kimpe, John E. Kraus, Michelle Eagle, Katie Rolfe, Laurent Servais, Joanna Nakielny, Afrodite Lourbakos, Michela Guglieri, Valeria Ricotti, Giles G Campion, Allison A Morgan, Francesco Muntoni, Volker Straub, Padraig Wright, Thomas Voit, Naashika Quarcoo
Publikováno v:
The Lancet Neurology. 13:987-996
Summary Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We invest
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4376b8b1e340ad1c94d88d59dcefb3a3
https://doi.org/10.1016/s1474-4422(14)70195-4
https://doi.org/10.1016/s1474-4422(14)70195-4