{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T18:49:10Z","timestamp":1793386150969,"version":"4.5.0"},"reference-count":15,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2018,5,29]],"date-time":"2018-05-29T00:00:00Z","timestamp":1527552000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2018,5,29]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Training methodology was established to optimize reliability of outcome measures in the nusinersen clinical trials. The Children\u2019s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb (RULM) were primary or secondary outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Video review, quarterly conference calls, and item scoring checks supported evaluator competence. Baseline and screening along with video review established intra and inter-rater reliability.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Inter and intra-rater reliability were both excellent. Intraclass correlation coefficients (ICC) ranged between 0.906\u20130.994 across initial training meetings and 0.824\u20130.996 across annual retraining meetings. This was similar for CHOP INTEND (ICC = 0.824\u20130.951), HFMSE (ICC = 0.981\u20130.996), and RULM (ICC = 0.966\u20130.990). Intra-rater reliability for the CHOP INTEND, HFMSE, and RULM were ICC = 0.895 (95% CI: 0.852\u20130.926; n = 116), ICC = 0.959 (95% CI: 0.942\u20130.971; n = 125), and ICC = 0.948 (95% CI: 0.927\u20130.963; n = 126) respectively.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Rigorous evaluator training ensures reliability of assessment of subjects with spinal muscular atrophy (SMA) in multicenter international trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-180301","type":"journal-article","created":{"date-parts":[[2018,5,29]],"date-time":"2018-05-29T11:23:55Z","timestamp":1527593035000},"page":"159-166","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":51,"title":["Evaluator Training and Reliability for SMA Global Nusinersen Trials"],"prefix":"10.1177","volume":"5","author":[{"given":"Allan M.","family":"Glanzman","sequence":"first","affiliation":[{"name":"Department of Physical Therapy, Children\u2019s Hospital of Philadelphia, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena S.","family":"Mazzone","sequence":"additional","affiliation":[{"name":"Department of Neurology, Catholic University, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sally Dunaway","family":"Young","sequence":"additional","affiliation":[{"name":"Department of Neurology, Columbia University, New York, NY, USA"},{"name":"Department of Rehabilitation andRegenerative Medicine, Columbia University, New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Gee","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy, Lucile Packard, Children\u2019s Hospital Stanford, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristy","family":"Rose","sequence":"additional","affiliation":[{"name":"Sydney University, Sydney, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"Newcastle University, Newcastle, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leslie","family":"Nelson","sequence":"additional","affiliation":[{"name":"UT Southwestern Medical Center, Dallas, TX, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Yun","sequence":"additional","affiliation":[{"name":"Lexa Enterprises, Inc., USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katie","family":"Alexander","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Basil T.","family":"Darras","sequence":"additional","affiliation":[{"name":"Department of Neurology, Boston Children\u2019s Hospital and Harvard Medical School, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zarazuela","family":"Zolkipli-Cunningham","sequence":"additional","affiliation":[{"name":"Division of Neurology, The Children\u2019s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gihan","family":"Tennekoon","sequence":"additional","affiliation":[{"name":"Division of Neurology, The Children\u2019s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John W.","family":"Day","sequence":"additional","affiliation":[{"name":"Department of Neurology, Stanford University School of Medicine, Stanford, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard S.","family":"Finkel","sequence":"additional","affiliation":[{"name":"Department of Neurology, NemoursChildren\u2019s Hospital, Orlando, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Department of Neurology, Catholic University, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Darryl C.","family":"De Vivo","sequence":"additional","affiliation":[{"name":"Department of Neurology, Columbia University, New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ron","family":"Baldwin","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathie M.","family":"Bishop","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacqueline","family":"Montes","sequence":"additional","affiliation":[{"name":"Department of Neurology, Columbia University, New York, NY, USA"},{"name":"Department of Rehabilitation andRegenerative Medicine, Columbia University, New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2018,5,29]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318271f7e4"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811420294"},{"key":"ref005","unstructured":"MazzoneES, MayhewA, MontesJ, RamseyD, FanelliL, Dunaway YoungS, et al. 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