Bibliografisk note

Funding Information:
GB has received speaker and consulting fees from Biogen, Novartis Gene Therapies, Inc. (AveXis), and Roche and has worked as principal investigator of SMA studies sponsored by Novartis Gene Therapies, Inc., and Roche. The other organizers do not have conflicts to declare for this specific workshop.We acknowledge especially the staff of the European Neuromuscular Center (ENMC) Alexandra Breukel, Managing Director and Annelies Zittersteijn, Operational Manager. This Workshop was made possible thanks to the financial support of the ENMC and ENMC main sponsors: Association Française contre les Myopathies (France), Deutsche Gesellschaft für Muskelkranke (Germany), Muscular Dystrophy Campaign (UK), Muskelsvindfonden (Denmark), Prinses Beatrix Spierfonds (The Netherlands), Schweizerische Stiftung für die Erforschung der Muskelkrankheiten (Switzerland), Telethon Foundation (Italy), Spierziekten Nederland (The Netherlands) and the associated members: Finnish Neuromuscular Association (Finland) and Österreichische Muskelforschung (Austria). The ENMC acknowledges the support of MDA USA and SMA Europe. Special thanks to the members of the ENMC Company Forum: Amicus Therapeutics, AveXis, Biogen, CSL Behring, Ionis Pharmaceuticals, PerkinElmer, Roche, Sanofi Genzyme, Sanquin Plasma Products, Santhera Pharmaceuticals, and other involved partner organizations for their support of the ENMC workshops. We thank SMA Europe and Cure SMA for the support provided. We acknowledge GB, PC, TG, ET, LvdP, BW have received funding from the European Union's Horizon 2020 research and innovation programme under the Marie Skłodowska-Curie Actions (MSCA) grant agreement SMABEYOND No 956185.

Funding Information:
We acknowledge especially the staff of the European Neuromuscular Center (ENMC) Alexandra Breukel, Managing Director and Annelies Zittersteijn, Operational Manager. This Workshop was made possible thanks to the financial support of the ENMC and ENMC main sponsors: Association Française contre les Myopathies (France), Deutsche Gesellschaft für Muskelkranke (Germany), Muscular Dystrophy Campaign (UK), Muskelsvindfonden (Denmark), Prinses Beatrix Spierfonds (The Netherlands), Schweizerische Stiftung für die Erforschung der Muskelkrankheiten (Switzerland), Telethon Foundation (Italy), Spierziekten Nederland (The Netherlands) and the associated members: Finnish Neuromuscular Association (Finland) and Österreichische Muskelforschung (Austria). The ENMC acknowledges the support of MDA USA and SMA Europe. Special thanks to the members of the ENMC Company Forum: Amicus Therapeutics, AveXis, Biogen, CSL Behring, Ionis Pharmaceuticals, PerkinElmer, Roche, Sanofi Genzyme, Sanquin Plasma Products, Santhera Pharmaceuticals, and other involved partner organizations for their support of the ENMC workshops. We thank SMA Europe and Cure SMA for the support provided. We acknowledge GB, PC, TG, ET, LvdP, BW have received funding from the European Union's Horizon 2020 research and innovation programme under the Marie Skłodowska-Curie Actions (MSCA) grant agreement SMABEYOND No 956185 .