Funding for this research was provided by:
Canadian Institutes of Health Research (Foundation Grant FDN-167281)
Ultragenyx Pharmaceutical
Canada Foundation for Innovation (CFI-JELF 38412)
Canadian Institutes of Health Research and Muscular Dystrophy Canada ((Network Catalyst Grant for NMD4C)
Canada Research Chairs program (950-232279)
Article History
Received: 23 October 2020
Accepted: 6 January 2021
First Online: 20 March 2021
Ethics approval and consent to participate
: Not applicable.
: Not applicable.
: HL—Consultancy and financial support for research projects and clinical trials from Amplo Biotechnology, AMO Pharma, Biogen, Desitin, Fulcrum Therapeutics, GW Pharma, Milo Biotechnology, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos and Ultragenyx. Editor-in-chief for the Journal of Neuromuscular Disease (IOS Press). ANR and EK are employees of and shareholders in Ultragenyx Pharmaceutical Inc.