{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,20]],"date-time":"2026-09-20T10:06:28Z","timestamp":1789898788189,"version":"4.0.1"},"reference-count":18,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2016,10,22]],"date-time":"2016-10-22T00:00:00Z","timestamp":1477094400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["J Child Neurol"],"published-print":{"date-parts":[[2017,2]]},"abstract":"<jats:p>The natural history of spinal muscular atrophy type I (SMA-I) has changed as improved medical support has become available. With investigational drugs for spinal muscular atrophy now in clinical trials, efficient trial design focuses on enrolling recently diagnosed infants, providing best available supportive care, and minimizing subject variation. The quandary has arisen whether it is ethically appropriate to specify a predefined level of nutritional and\/or ventilation support for spinal muscular atrophy type I subjects while participating in these studies. We conducted a survey at 2 spinal muscular atrophy investigator meetings involving physician investigators, clinical evaluators, and study coordinators from North America, Europe, and Asia-Pacific. Each group endorsed the concept that having a predefined degree of nutritional and ventilation support was warranted in this context. We discuss how autonomy, beneficence\/non-maleficence, noncoercion, social benefit, and equipoise can be maintained when a predefined level of supportive care is proposed, for participation in a clinical trial.<\/jats:p>","DOI":"10.1177\/0883073816671236","type":"journal-article","created":{"date-parts":[[2016,10,19]],"date-time":"2016-10-19T22:24:01Z","timestamp":1476915841000},"page":"155-160","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":18,"title":["Spinal Muscular Atrophy Type I"],"prefix":"10.1177","volume":"32","author":[{"given":"Richard S.","family":"Finkel","sequence":"first","affiliation":[{"name":"Division of Neurology, Nemours Children\u2019s Hospital, Orlando, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathie M.","family":"Bishop","sequence":"additional","affiliation":[{"name":"Tioga Pharmaceuticals, San Diego, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert M.","family":"Nelson","sequence":"additional","affiliation":[{"name":"US Food and Drug Administration, Silver Spring, MD, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2016,10,22]]},"reference":[{"key":"bibr1-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.1995.00540290108025"},{"key":"bibr2-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000290830.40544.b9"},{"key":"bibr3-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1086\/338627"},{"key":"bibr4-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"bibr5-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1002\/ana.20473"},{"key":"bibr6-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305788"},{"key":"bibr7-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.201207087"},{"key":"bibr8-0883073816671236","first-page":"99","volume":"6","author":"Chopra JS","year":"1991","journal-title":"Crit Rev Neurobiol"},{"key":"bibr9-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.12291"},{"key":"bibr10-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpedsurg.2008.05.035"},{"key":"bibr11-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1136\/adc.2009.166512"},{"key":"bibr12-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1016\/j.neurol.2012.07.020"},{"key":"bibr13-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1016\/j.prrv.2006.04.200"},{"key":"bibr14-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.04.009"},{"key":"bibr15-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-642-20195-0_11"},{"key":"bibr16-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1017\/S096318011400053X"},{"key":"bibr17-0883073816671236","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305788"},{"key":"bibr18-0883073816671236","unstructured":"European Neuromuscular Centre, 218th ENMC International Workshop: \u201cEight years on, revisiting the Consensus Statement of Care in SMA,\u201d February 19-21, 2016. enmc.org."}],"container-title":["Journal of Child Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/0883073816671236","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/0883073816671236","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/0883073816671236","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T20:38:46Z","timestamp":1777495126000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/0883073816671236"}},"subtitle":["Is It Ethical to Standardize Supportive Care Intervention in Clinical Trials?"],"short-title":[],"issued":{"date-parts":[[2016,10,22]]},"references-count":18,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2017,2]]}},"alternative-id":["10.1177\/0883073816671236"],"URL":"https:\/\/doi.org\/10.1177\/0883073816671236","relation":{},"ISSN":["0883-0738","1708-8283"],"issn-type":[{"value":"0883-0738","type":"print"},{"value":"1708-8283","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,10,22]]}}}