Johnson, Katherine
Töpf, Ana
Bertoli, Marta
Phillips, Lauren
Claeys, Kristl G.
Stojanovic, Vidosava Rakocevic
Perić, Stojan
Hahn, Andreas
Maddison, Paul
Akay, Ela
Bastian, Alexandra E.
Łusakowska, Anna
Kostera-Pruszczyk, Anna
Lek, Monkol
Xu, Liwen
MacArthur, Daniel G.
Straub, Volker
Funding for this research was provided by:
Sanofi Genzyme
Ultragenyx
Kurt+Peter Foundation
LGMD2I Research Fund
Samantha J Brazzo Foundation
LGMD2D Foundation
Muscular Dystrophy UK
Coalition to Cure Calpain 3
National Human Genome Research Institute (UM1 HG008900, UM1 HG008900)
National Eye Institute (UM1 HG008900, UM1 HG008900)
National Heart, Lung, and Blood Institute (UM1 HG008900, UM1 HG008900)
MDA/AANEM Development Grant
Ministarstvo Prosvete, Nauke i Tehnološkog Razvoja (175083)
Article History
Received: 12 June 2017
Accepted: 6 November 2017
First Online: 17 November 2017
Ethics approval and consent to participate
: Full ethics approval was granted for the Newcastle MRC Centre Biobank for Rare and Neuromuscular Diseases by the Newcastle and North Tyneside research ethics committee (REC reference number 09/H0906/28). All patients provided informed consent for the storage and use of their biomaterial in the Newcastle MRC Centre Biobank for Rare and Neuromuscular Diseases.
: Not applicable.
: VS is or has been a principal investigator for trials sponsored by Sanofi Genzyme, GSK, Prosensa/BioMarin Pharmaceuticals, Ionis Pharmceuticals and Sarepta Therapeutics. VS received speaker honoraria from Sanofi Genzyme. For the last 3 years VS is or has been on advisory boards for Audentes Therapeutics, Biogen, Bristol-Myer Squibb, Italfarmaco S.p.A., Sarepta Therapeutics, Summit Therapeutics, Tivorsan Pharmaceuticals, TrophyNOD, and Wave Therapeutics. KGC is principal investigator for trials sponsored by Sanofi Genzyme and KGC received speaker honoraria and travel grants from Sanofi Genzyme. The authors’ declare that they have no competing interests.
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