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Elkins, Sai Dharmarajan, Katherine Gooch, Ivana Audhya: Employees of Sarepta Therapeutics, Inc., and may hold stock\/options in the company. Teofil Ciobanu: Employee of F. Hoffmann-La Roche Ltd. Claire J. Lansdall: Employee of F. Hoffmann-La Roche Ltd. and shareholder of F. Hoffmann-La Roche Ltd. Alexander P. Murphy: Employee of Roche Products UK and may hold shares in F. Hoffmann-La Roche Ltd. Fiona McDougall: Employee of Genentech, Inc.and shareholder of F. Hoffmann-La Roche Ltd. Craig M. McDonald: Reports grants from Capricor Therapeutics, Catabasis, Edgewise Therapeutics, Epirium Bio, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics; and has a consultancy\/advisory role with Biomarin, Capricor Therapeutics, Catalyst, Edgewise Therapeutics, Italfarmaco, PTC Therapeutics, F. Hoffmann-La Roche Ltd, Santhera Pharmaceuticals and Sarepta Therapeutics. He has received honoraria from PTC Therapeutics and Sarepta Therapeutics. Eugenio M. Mercuri: Reports receiving fees from AveXis, Biogen, and F. Hoffmann-La Roche Ltd.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"EMBARK was conducted in accordance with the provisions of the Declaration of Helsinki and Good Clinical Practice guidelines. Trial protocol and all amendments were approved by an institutional review board and ethics committee at each site. The full list of institutional review boards and ethics committees is available in the Supplementary Information. Informed consent, including consent to publish, was obtained from parent(s)\/legal guardian(s), and patients\u2019 assent was obtained when indicated.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}