Funding for this research was provided by:
Limb Girdle Muscular Dystrophy 2i Research Fund
Muscular Dystrophy UK
National Human Genome Research Institute (UM1 HG008900)
National Eye Institute (UM1 HG008900)
National Heart, Lung, and Blood Institute (UM1 HG008900)
Samantha J Brazzo Foundation
Coalition to Cure Calpain 3
MDA/AANEM Development Grant
Ministarstvo Prosvete, Nauke i Tehnološkog Razvoja (175083)
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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