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She also received travel expenses from Biogen. PS, SW, KW and MRAS report no disclosures. RG has received honoraria from Biogen as an Advisory board member. JCK has received financial research support form TEVA Pharmaceuticals and honoraria as speaker\/consultant for AbbVie, Allergan, Biogen, Ipsen and AveXis\/Novartis. PL has received financial research support from TEVA Pharmaceuticals and honoraria as speaker\/consultant for AbbVie, Atheneum Partners, BIAL, Desitin, Licher MT, Medtronic, Novartis. ZU has received honoraria from Biogen as a consultant. BW has received honoraria from Biogen for a lecture. AO has received honoraria from Biogen as a consultant. OSK has received honoraria as a speaker\/consultant and\/or funding for travel expenses from the Deutsche Gesellschaft f\u00fcr Muskelkranke (DGM e.V.), Novartis, Biogen GmbH, the Jain Foundation. She received academic research support by Ludwig-Maximilians-University of Munich, FoeFoLe program, Reg.-No. 747, 11\/11\u201305\/13, and MHH Young Faculty Program, 2018\u20132020 as well as research support by foundations and societies by the Friedrich-Baur-GmbH, Burgkunstadt 12\/13\u201312\/15 and the Deutsche Gesellschaft f\u00fcr Muskelkranke (DGM e.V.), 2019\u201320. ACL received financial research support from AB Science, Biogen Idec, Cytokinetics, GSK, Orion Pharma, Novartis, TauRx Therapeutics Ltd. and TEVA Pharmaceuticals. He also has received honoraria as a consultant from Mitsubishi, Orion Pharma, Novartis, Teva and as an Advisory board member of Biogen and Hoffmann-La Roche. SP has received honoraria as speaker\/consultant from Biogen Idec, Novartis, Cytokinetics, TEVA Pharmaceuticals, Desitin. AH has received honoraria from Biogen and Desitin as a consultant. MO received honoraria as consultant from Biogen, Axon and Fujirebio.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"The study was approved by the local ethics committees of the centers involved in Ulm, Dresden, G\u00f6ttingen and Hannover (approval number at central study center at Ulm University 19\/12; 2012) and all patients or their relatives (legal guardian) gave informed written consent to participate in the study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical standards"}}]}}