Preclinical Toxicological Assessment of A Novel siRNA, SLN360, Targeting Elevated Lipoprotein (a) in Cardiovascular Disease

por Rider, David, Chivers, Simon, Aretz, Julia, Eisermann, Mona, Löffler, Kathrin, Hauptmann, Judith, Morrison, Eliot, Campion, Giles
Publicado 2022

Treatment and prevention of lipoprotein(a)-mediated cardiovascular disease: the emerging potential of RNA interference therapeutics

por Swerdlow, Daniel I, Rider, David A, Yavari, Arash, Wikström Lindholm, Marie, Campion, Giles V, Nissen, Steven E
Publicado 2021

Exon skipping for DMD

por Aartsma-Rus, Annemieke, Verschuuren, Jan JGM, Campion, Giles V, van Ommen, Gert-jan B, van Deutekom, Judith CT
Publicado 2012

Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

por McDonald, Craig M., Wong, Brenda, Flanigan, Kevin M., Wilson, Rosamund, de Kimpe, Sjef, Lourbakos, Afrodite, Lin, Zhengning, Campion, Giles
Publicado 2018

A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy

por Beekman, Chantal, Sipkens, Jessica A., Testerink, Janwillem, Giannakopoulos, Stavros, Kreuger, Dyonne, van Deutekom, Judith C., Campion, Giles V., de Kimpe, Sjef J., Lourbakos, Afrodite
Publicado 2014

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

por Goemans, Nathalie M., Tulinius, Már, van den Hauwe, Marleen, Kroksmark, Anna-Karin, Buyse, Gunnar, Wilson, Rosamund J., van Deutekom, Judith C., de Kimpe, Sjef J., Lourbakos, Afrodite, Campion, Giles
Publicado 2016

Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial

por Flanigan, Kevin M., Voit, Thomas, Rosales, Xiomara Q., Servais, Laurent, Kraus, John E., Wardell, Claire, Morgan, Allison, Dorricott, Susie, Nakielny, Joanna, Quarcoo, Naashika, Liefaard, Lia, Drury, Tom, Campion, Giles, Wright, Padraig
Publicado 2013

Dystrophin quantification: Biological and translational research implications

por Anthony, Karen, Arechavala-Gomeza, Virginia, Taylor, Laura E., Vulin, Adeline, Kaminoh, Yuuki, Torelli, Silvia, Feng, Lucy, Janghra, Narinder, Bonne, Gisèle, Beuvin, Maud, Barresi, Rita, Henderson, Matt, Laval, Steven, Lourbakos, Afrodite, Campion, Giles, Straub, Volker, Voit, Thomas, Sewry, Caroline A., Morgan, Jennifer E., Flanigan, Kevin M., Muntoni, Francesco
Publicado 2014