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Neurol Sci. 2019;40(2):327\u201332.","journal-title":"Neurol Sci"}],"container-title":["CNS Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40263-019-00656-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40263-019-00656-w\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40263-019-00656-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,8,10]],"date-time":"2021-08-10T17:51:15Z","timestamp":1628617875000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40263-019-00656-w"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,8,16]]},"references-count":50,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2019,9]]}},"alternative-id":["656"],"URL":"https:\/\/doi.org\/10.1007\/s40263-019-00656-w","relation":{},"ISSN":["1172-7047","1179-1934"],"issn-type":[{"value":"1172-7047","type":"print"},{"value":"1179-1934","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,8,16]]},"assertion":[{"value":"16 August 2019","order":1,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Compliance with Ethical Standards"}},{"value":"All study designs were approved by institutional review boards at study sites and were conducted in accordance with Good Clinical Practice guidelines.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}},{"value":"Written informed consent was obtained from parents or legal guardians, and based on institutional guidelines and the child\u2019s age, participants provided assent as appropriate.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed Consent"}},{"value":"These studies were sponsored by Ionis Pharmaceuticals, Inc. (Carlsbad, CA, USA) and Biogen (Cambridge, MA, USA). Biogen also provided funding for the open access fee and medical writing support in the development of this paper. Meryl Mandle and Allison Green from Excel Scientific Solutions wrote the first draft of the manuscript based on input from authors, and Nathaniel Hoover from Excel Scientific Solutions copyedited and styled the manuscript per the journal requirements. Biogen and Ionis Pharmaceuticals, Inc. reviewed and provided feedback on the manuscript to the authors. The authors had full editorial control of the manuscript and provided their final approval of all content.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"Basil T. Darras has served on advisory boards for AveXis, Biogen, Cytokinetics, Dynacure, Genentech, PTC, Roche, and Sarepta; served on a speakers bureau for Biogen; received research support from the National Institutes of Health\/National Institute of Neurological Disorders and Stroke, the Slaney Family Fund for SMA, the SMA Foundation, and the Working on Walking Fund; received grants from Biogen, CureSMA, and Ionis Pharmaceuticals, Inc. during ENDEAR, CHERISH, CS2, CS11, and CS12; received grants from Cytokinetics, FibroGen, PTC, Roche, Santhera, Sarepta, and Summit; and has no personal financial interests in these companies. Michelle A. Farrar has received scientific advisory board honoraria from Biogen and Roche; is a principal investigator for ongoing AveXis and Biogen clinical trials; and has received funding from Motor Neuron Diseases Research Institute of Australia. Eugenio Mercuri has served on advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Inc., Novartis, and Roche; is a principal investigator for ongoing Ionis Pharmaceuticals, Inc.\/Biogen and Roche clinical trials; and has received funding from Famiglie SMA Italy, Italian Telethon, and SMA Europe. Richard S. Finkel has received grants and advisor fees from Biogen and Ionis Pharmaceuticals, Inc. during ENDEAR and CHERISH; received grants from AveXis, Cytokinetics, and Roche; served as an advisor to AveXis, Novartis, and Roche outside the submitted work; served in an advisory capacity to nonprofit organizations CureSMA, SMA Europe, the SMA Foundation, and SMA Reach (UK); and served on a data safety monitoring board for the AveXis AVXS-101 phase 1 gene transfer study and the Roche Moonfish phase 1b study; received fees for presentations at CME activities sponsored by AveXis and Biogen; and has received licensing fees from the Children\u2019s Hospital of Philadelphia for co-creating the CHOP INTEND motor scale for SMA. Richard Foster is an employee of and stockholder in Biogen. Steven G. Hughes was an employee of and holds stock\/stock options in Ionis Pharmaceuticals, Inc. He now consults for Ionis Pharmaceuticals, Inc. Ishir Bhan is an employee of and stockholder in Biogen. Wildon Farwell is an employee of and stockholder in Biogen. Sarah Gheuens is an employee of and stockholder in Biogen.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}