Johnson, Katherine
Bertoli, Marta
Phillips, Lauren
Töpf, Ana
Van den Bergh, Peter
Vissing, John
Witting, Nanna
Nafissi, Shahriar
Jamal-Omidi, Shirin
Łusakowska, Anna
Kostera-Pruszczyk, Anna
Potulska-Chromik, Anna
Deconinck, Nicolas
Wallgren-Pettersson, Carina
Strang-Karlsson, Sonja
Colomer, Jaume
Claeys, Kristl G.
De Ridder, Willem
Baets, Jonathan
von der Hagen, Maja
Fernández-Torrón, Roberto
Zulaica Ijurco, Miren
Espinal Valencia, Juan Bautista
Hahn, Andreas
Durmus, Hacer
Willis, Tracey
Xu, Liwen
Valkanas, Elise
Mullen, Thomas E.
Lek, Monkol
MacArthur, Daniel G.
Straub, Volker
Funding for this research was provided by:
Sanofi Genzyme
Ultragenyx
LGMD2I Research Fund
Samantha J Brazzo Foundation
LGMD2D Foundation
Kurt+Peter Foundation
Muscular Dystrophy UK
Coalition to Cure Calpain 3
National Human Genome Research Institute (UM1 HG008900)
Article History
Received: 25 April 2018
Accepted: 13 July 2018
First Online: 30 July 2018
Ethics approval and consent to participate
: Ethical approval (REC reference number 08/H0906/28) was granted by the Newcastle and North Tyneside Research Ethics Committee. All patients provided informed written consent to participate in the project.
: Not applicable.
: VS is or has been on advisory boards for Acceleron Pharma, Audentes Therapeutics, Biogen, Biomarin, Bristol-Myer Squibb, Italfarmaco S.p.A., Nicox, Pfizer, Sanofi Genzyme, Santhera Pharmaceuticals, Sarepta Therapeutics, Summit Therapeutics, Tivorsan, TrophyNOD, and Wave Therapeutics. VS received speaker honoraria from Sanofi Genzyme. VS has or had research collaborations with Ultragenyx Pharmaceuticals and Sanofi Genzyme. All other authors declare that they have no competing interests.
: Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.