Zobrazeno 1 - 10
of 11
pro vyhledávání: '"Erik, Niks"'
Autor:
Mirko Signorelli, Burcu Ayoglu, Camilla Johansson, Hanns Lochmüller, Volker Straub, Francesco Muntoni, Erik Niks, Roula Tsonaka, Anja Persson, Annemieke Aartsma‐Rus, Peter Nilsson, Cristina Al‐Khalili Szigyarto, Pietro Spitali
Publikováno v:
Journal of Cachexia, Sarcopenia and Muscle, Vol 11, Iss 2, Pp 505-517 (2020)
Abstract Background Duchenne muscular dystrophy (DMD) is a fatal disease for which no cure is available. Clinical trials have shown to be largely underpowered due to inter‐individual variability and noisy outcome measures. The availability of bioma
Externí odkaz:
https://doaj.org/article/279f86f1eb524b11a1cf2dcdaf841012
Autor:
Anna Sarkozy, Rosaline Quinlivan, John P. Bourke, Alessandra Ferlini, Inès Barthélémy, Linda H Cripe, Emily Reuben, Teresinha Evangelista, Anca Florian, Josh Gribnau, Lidia Gonzalez-Quereda, Michela Guglieri, Erik Niks, Rahul Phadke, Luisa Politano, Ros Quinlivan, John Vissing, Nicol Voermans, Elizabeth Vroom, Aleksandra Pietrusz, Fernanda Fortunato, Saskia Houwen
Publikováno v:
Neuromuscular Disorders, 33, 3, pp. 274-284
Neuromuscular Disorders, 33(3), 274-284. Elsevier Ltd.
Neuromuscular Disorders, 33, 274-284
Neuromuscular Disorders, 33(3), 274-284. Elsevier Ltd.
Neuromuscular Disorders, 33, 274-284
Item does not contain fulltext
Autor:
Giorgia Coratti, Marika Pane, Claudia Brogna, Valeria Ricotti, Sonia Messina, Adele D'Amico, Claudio Bruno, Gianluca Vita, Angela Berardinelli, Elena Mazzone, Francesca Magri, Federica Ricci, Tiziana Mongini, Roberta Battini, Luca Bello, Elena Pegoraro, Giovanni Baranello, Stefano C Previtali, Luisa Politano, Giacomo P Comi, Valeria A Sansone, Alice Donati, Jean Yves Hogrel, Volker Straub, Silvana De Lucia, Erik Niks, Laurent Servais, Imelda De Groot, Mary Chesshyre, Enrico Bertini, Nathalie Goemans, Francesco Muntoni, Eugenio Mercuri, on behalf on the International DMD Group and the iMDEX Consortium
Publikováno v:
PLoS ONE, Vol 16, Iss 6, p e0253882 (2021)
IntroductionThe aim of this study was to report 36-month longitudinal changes using the North Star Ambulatory Assessment (NSAA) in ambulant patients affected by Duchenne muscular dystrophy amenable to skip exons 44, 45, 51 or 53.Materials and methods
Externí odkaz:
https://doaj.org/article/be2f7562560244a9a2a56400fc1fd6e8
Autor:
Camilla Johansson, Helian Hunt, Mirko Signorelli, Fredrik Edfors, Andreas Hober, Anne-Sophie Svensson, Hanna Tegel, Björn Forstström, Annemieke Aartsma-Rus, Erik Niks, Pietro Spitali, Mathias Uhlén, Cristina Al-Khalili Szigyarto
Background: Molecular components in blood, like proteins, are used as biomarkers to reveal or predict disease states, guide clinical interventions and aid development of therapies. While multiplexing proteomics methods promote discovery of such bioma
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::5cb672e9c3af49befb6244d885e65dea
https://doi.org/10.21203/rs.3.rs-2285032/v1
https://doi.org/10.21203/rs.3.rs-2285032/v1
Autor:
Nienke M. Van de Velde, Jurriaan De Groot, Erik Niks, Melissa Hooijmans, Thom Veeger, Kevin Keene, Andrew Webb, Hermien Kan
Publikováno v:
NMR in Biomedicine, 35(7). WILEY
NMR in biomedicine, 35(7):e4691. John Wiley and Sons Ltd
NMR in biomedicine, 35(7):e4691. John Wiley and Sons Ltd
In Becker muscular dystrophy (BMD), muscle weakness progresses relatively slowly, with a highly variable rate among patients. This complicates clinical trials, as clinically relevant changes are difficult to capture within the typical duration of a t
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::82260761046e46703156a090db8abb84
http://hdl.handle.net/1887/3276366
http://hdl.handle.net/1887/3276366
Autor:
Victor, Ronald G, Sweeney, H. Lee, Finkel, Richard, Mcdonald, Craig M, Byrne, Barry, Eagle, Michelle, Goemans, Nathalie, Vandenborne, Krista, Dubrovsky, Alberto L, Topaloglu, Haluk, Miceli, M. Carrie, Furlong, Pat, Landry, John, Elashoff, Robert, Cox, David, Hoda, Abdel-Hamid, Susan, Apkon, Richard, Barohn, Elena, Belousova, Enrico, Bertini, John, Brandsema, Claudio, Bruno, William, Burnette, Russell, Butterfield, Barry, Byrne, Craig, Campbell, Jose, Carlo, Jong-Hee, Chae, Saleel, Chandratre, Giacomo, Comi, Anne, Connolly, Imelda De Groot, Nicolas, Deconinck, Joseph, Dooley, Alberto, Dubrovsky, Julien, Durigneux, Erika, Finanger, Richard, Finkel, L Matthew Frank, Nathalie, Goemans, Amy, Harper, Ayako, Hattori, Ozlem, Herguner, Susan, Iannaccone, Joanne, Janas, Yuh-Jyh, Jong, Janberd, Kirschner, Hirofumi, Komaki, Nancy, Kuntz, Wang-Tso, Lee, Edward, Leung, Jean, Mah, Katherine, Mathews, Craig, Mcdonald, Eugenio, Mercuri, Hugh, Mcmillan, Wolfgang, Mueller-Felber, Adolfo Lopez de Munain, Akinori, Nakamura, Erik, Niks, Katsuhisa, Ogata, Samuel, Pascual, Pegoraro, Elena, Yann, Pereon, Ben, Renfroe, Ratna Bhavaraju Sanka, Jens, Schallner, Ulrike, Schara, Kathryn, Selby, Isabel Illa Sendra, Laurent, Servais, Edward, Smith, Susan, Sparks, Haluk, Topaloglu, Ron, Victor, Juan Jose Vilchez, Matthew, Wicklund, Ekkehard, Wilichoswki, Brenda, Wong
Publikováno v:
Neurology, vol 89, iss 17
Neurology
Neurology, 89(17), 1811-1820
Neurology, 89 (17
Neurology
Neurology, 89(17), 1811-1820
Neurology, 89 (17
To conduct a randomized trial to test the primary hypothesis that once-daily tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD).
info:eu-repo/semantics/published
info:eu-repo/semantics/published
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6e0e6503a882dfb553f43c58e0475c30
https://ora.ox.ac.uk/objects/uuid:07acefc6-f3c1-43d6-aaff-dd314f53221c
https://ora.ox.ac.uk/objects/uuid:07acefc6-f3c1-43d6-aaff-dd314f53221c
Autor:
Pinki Munot, Stephanie A. Robb, Erik H. Niks, Jacqueline Palace, Erik Niks, Stephanie Robb, Amelia Evoli, Andrea Klein, Pedro Rodriquez Cruz, Bruno Eymard, Heinz Jungbluth, Corrie Erasmus, Adela Della Marina, Fulvio Baggi, Nancy Kuntz, Malene Børresen, Imelda Hughes, Sithara Ramdas, Monique Ryan, Matthew Pitt
Publikováno v:
Neuromuscular Disorders, 30(3), 254-264. PERGAMON-ELSEVIER SCIENCE LTD
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::87cd90b4d0d59a80d83e00420c431f01
https://hdl.handle.net/1887/3182689
https://hdl.handle.net/1887/3182689
Autor:
Sara Baldassari, Fabienne Picard, Nienke E. Verbeek, Marjan van Kempen, Eva H. Brilstra, Gaetan Lesca, Valerio Conti, Renzo Guerrini, Francesca Bisulli, Laura Licchetta, Tommaso Pippucci, Paolo Tinuper, Edouard Hirsch, Anne de Saint Martin, Jamel Chelly, Gabrielle Rudolf, Mathilde Chipaux, Sarah Ferrand-Sorbets, Georg Dorfmüller, Sanjay Sisodiya, Simona Balestrini, Natasha Schoeler, Laura Hernandez-Hernandez, S. Krithika, Renske Oegema, Eveline Hagebeuk, Boudewijn Gunning, Charles Deckers, Bianca Berghuis, Ilse Wegner, Erik Niks, Floor Jansen, Kees Braun, Daniëlle de Jong, Guido Rubboli, Inga Talvik, Valentin Sander, Peter Uldall, Marie-Line Jacquemont, Caroline Nava, Eric Leguern, Sophie Julia, Antonio Gambardella, Giuseppe d’Orsi, Giovanni Crichiutti, Laurence Faivre, Veronique Darmency, Barbora Benova, Pavel Krsek, Arnaud Biraben, Anne-Sophie Lebre, Mélanie Jennesson, Shifteh Sattar, Cécile Marchal, Douglas R. NordliJr, Kristin Lindstrom, Pasquale Striano, Lysa Boissé Lomax, Courtney Kiss, Fabrice Bartolomei, Anne Fabienne Lepine, An-Sofie Schoonjans, Katrien Stouffs, Anna Jansen, Eleni Panagiotakaki, Brigitte Ricard-Mousnier, Julien Thevenon, Julitta de Bellescize, Hélène Catenoix, Thomas Dorn, Martin Zenker, Karen Müller-Schlüter, Christian Brandt, Ilona Krey, Tilman Polster, Markus Wolff, Meral Balci, Kevin Rostasy, Guillaume Achaz, Pia Zacher, Thomas Becher, Thomas Cloppenborg, Christopher J. Yuskaitis, Sarah Weckhuysen, Annapurna Poduri, Johannes R. Lemke, Rikke S. Møller, Stéphanie Baulac
Publikováno v:
Genetics in Medicine
The original version of this Article contained an error in the author list where the corresponding author Stéphanie Baulac was repeated twice. This has now been corrected in the HTML, the PDF was correct at the time of publication.
Autor:
Annemieke Aartsma-Rus, Pat Furlong, Elizabeth Vroom, Gert-Jan van Ommen, Erik Niks, Chiara Straathof, Jan Verschuuren, Alessandra Ferlini, Lynn Hagger, Emma Heslop, Veronika Karcagi, Jan Kirschner, Pauline McCormack, Pat Moeschen, Francesco Muntoni, Marie-Christine Ouillade, Jes Rahbeck, Christoph Rehmann-Sutter, Francoise Rouault, Tomas Sejersen, Simon Woods
Publikováno v:
Neuromuscular disorders : NMD. 21(1)
There is currently no cure for Duchenne muscular dystrophy, but there are some promising treatments in development, of which antisense-mediated exon skipping is close to clinical application. The results of the first trials have resulted in significa
Autor:
Annemieke Aartsma-Rus, Pat Furlong, Elizabeth Vroom, Gert-Jan van Ommen, Erik Niks, Chiara Straathof, Jan Verschuuren, Alessandra Ferlini, Lynn Hagger, Emma Heslop, Veronika Karcagi, Jan Kirschner, Pauline McCormack, Pat Moeschen, Francesco Muntoni, Marie-Christine Ouillade, Jes Rahbeck, Christoph Rehmann-Sutter, Francoise Rouault, Tomas Sejersen, Simon Woods
Publikováno v:
Neuromuscular Disorders. 21:527-528