{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T22:06:47Z","timestamp":1790892407649,"version":"4.1.0"},"reference-count":61,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2013,7,26]],"date-time":"2013-07-26T00:00:00Z","timestamp":1374796800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2014,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:p><jats:italic>Introduction<\/jats:italic>: Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). <jats:italic>Methods<\/jats:italic>: Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. <jats:italic>Results<\/jats:italic>: Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. <jats:italic>Conclusions<\/jats:italic>: The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges. <jats:italic>Muscle Nerve<\/jats:italic> <jats:bold>49<\/jats:bold>:422\u2013430, 2014<\/jats:p>","DOI":"10.1002\/mus.23937","type":"journal-article","created":{"date-parts":[[2013,7,8]],"date-time":"2013-07-08T12:08:39Z","timestamp":1373285319000},"page":"422-430","source":"Crossref","is-referenced-by-count":71,"title":["Rasch analysis of clinical outcome measures in spinal muscular atrophy"],"prefix":"10.1002","volume":"49","author":[{"given":"Stefan J.","family":"Cano","sequence":"first","affiliation":[{"name":"Clinical Neurology Research Group, Room N13 ITTC Building Peninsula College of Medicine and Dentistry Tamar Science Park Plymouth United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"Newcastle Muscle Team Institute of Human Genetics, International Centre for Life Central Parkway Newcastle United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allan M.","family":"Glanzman","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy The Children's Hospital of Philadelphia Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin J.","family":"Krosschell","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy and Human Movement Sciences Feinberg School of Medicine Northwestern University Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn J.","family":"Swoboda","sequence":"additional","affiliation":[{"name":"University of Utah School of Medicine Salt Lake City Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marion","family":"Main","sequence":"additional","affiliation":[{"name":"Great Ormond Street Children's Hospital for the Dubowitz Neuromuscular Center London England"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Birgit F.","family":"Steffensen","sequence":"additional","affiliation":[{"name":"Rehabiliterings Center for Muskelsvind Aarhus Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carole","family":"B\u00e9rard","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Lyon Sud France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fran\u00e7oise","family":"Girardot","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Lyon Sud France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine A.M.","family":"Payan","sequence":"additional","affiliation":[{"name":"Department de Pharmacologie Clinique H\u00f4pital Piti\u00e9\u2010Salp\u00eatri\u00e8re Assistance Publique\u2010H\u00f4pitaux de Paris (AP\u2010HP) Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology Neuropsichiatra Infantile Polclinico Gemell, Largo Gemelli Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Mazzone","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology Neuropsichiatra Infantile Polclinico Gemell, Largo Gemelli Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bakri","family":"Elsheikh","sequence":"additional","affiliation":[{"name":"Division of Neuromuscular Medicine, Department of Neurology The Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julaine","family":"Florence","sequence":"additional","affiliation":[{"name":"Neuromuscular Division; Department of Neurology Washington University School of Medicine St. Louis Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda S.","family":"Hynan","sequence":"additional","affiliation":[{"name":"Departments of Clinical Sciences (Biostatistics) and Psychiatry University of Texas Southwestern Medical Center Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan T.","family":"Iannaccone","sequence":"additional","affiliation":[{"name":"Pediatrics and Neurology and Neurotherapeutics University of Texas Southwestern Medical Center Pediatric Neurology, Children's Medical Center Ambulatory Care Pavilion Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leslie L.","family":"Nelson","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy University of Texas Southwestern Medical Center Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shree","family":"Pandya","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Rochester School of Medicine and Dentistry Rochester New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Rose","sequence":"additional","affiliation":[{"name":"Department of Neurology King's College Hospital Denmark Hill London United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles","family":"Scott","sequence":"additional","affiliation":[{"name":"CBS Squared, Inc Fort Washington, PA for Project Cure  USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reza","family":"Sadjadi","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Virginia Charlottesville Virginia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mackensie A.","family":"Yore","sequence":"additional","affiliation":[{"name":"Spinal Muscular Atrophy Foundation New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cynthia","family":"Joyce","sequence":"additional","affiliation":[{"name":"Spinal Muscular Atrophy Foundation New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John T.","family":"Kissel","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics The Ohio State University\/Nationwide Children's Hospital 395 W. 12th Avenue Columbus Ohio 43210 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the International Coordinating Committee for SMA Clinical Trials Rasch Task Force","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2013,7,26]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/ejhg.2011.134"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuron.2005.12.001"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.1995.00540290108025"},{"key":"e_1_2_7_5_1","first-page":"1","article-title":"Drug treatment for spinal muscular atrophy Type I","volume":"1","author":"Bosboom W","year":"2009","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_2_7_6_1","first-page":"1","article-title":"Drug treatment for spinal muscular atrophy types II and III","volume":"1","author":"Bosboom W","year":"2009","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2011.74"},{"key":"e_1_2_7_8_1","unstructured":"Food and Drug Administration.Patient reported outcome measures: use in medical product development to support labelling claims [online]. Available at:http:\/\/www.fda.gov\/downloads\/Drugs\/Guidances\/UCM193282.pdf."},{"key":"e_1_2_7_9_1","unstructured":"Food and Drug Administration.Qualification process for drug development tools [online]. Available at:www.fda.gov\/cber\/gdlns\/prolbl.pdf.http:\/\/www.fda.gov\/downloads\/Drugs\/GuidanceComplianceRegulatoryInformation\/Guidances\/UCM193282.pdf."},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1749-6632.1998.tb11015.x"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.3310\/hta13120"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318271f7e4"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1090-3798(03)00060-6"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2005.11.010"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811420294"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2006.03.015"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.22040"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2005.03.004"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2006.03.005"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.60.8.1130"},{"key":"e_1_2_7_21_1","volume-title":"32nd Annual Meeting of British Paediatric Neurology Association","author":"Scott E","year":"2006"},{"key":"e_1_2_7_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.06.368"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/pri.221"},{"key":"e_1_2_7_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_2_7_25_1","doi-asserted-by":"publisher","DOI":"10.1097\/PEP.0b013e3182351f04"},{"key":"e_1_2_7_26_1","doi-asserted-by":"publisher","DOI":"10.1097\/PEP.0b013e318181ae96"},{"key":"e_1_2_7_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2007.05.009"},{"key":"e_1_2_7_28_1","volume-title":"Gross motor function measure user's manual","author":"Russell D","year":"2002"},{"key":"e_1_2_7_29_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2002.tb00847.x"},{"key":"e_1_2_7_30_1","doi-asserted-by":"publisher","DOI":"10.1097\/PEP.0b013e31815f66a6"},{"key":"e_1_2_7_31_1","doi-asserted-by":"publisher","DOI":"10.4135\/9781412985598"},{"key":"e_1_2_7_32_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000142962.11881.26"},{"key":"e_1_2_7_33_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.22420"},{"key":"e_1_2_7_34_1","doi-asserted-by":"publisher","DOI":"10.3310\/hta13120"},{"key":"e_1_2_7_35_1","doi-asserted-by":"publisher","DOI":"10.1586\/erp.11.59"},{"key":"e_1_2_7_36_1","volume-title":"Probabilistic models for some intelligence and attainment tests","author":"Rasch G","year":"1980"},{"key":"e_1_2_7_37_1","doi-asserted-by":"publisher","DOI":"10.1186\/1477-7525-6-58"},{"key":"e_1_2_7_38_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2011.03939.x"},{"key":"e_1_2_7_39_1","volume-title":"Meeting of the FDA SEALD Division and Representatives of the International Coordinating Committee for SMA Clinical Trials","author":"International Coordinating Committee for SMA Clinical Trials","year":"2010"},{"key":"e_1_2_7_40_1","doi-asserted-by":"publisher","DOI":"10.3109\/09286586.2010.545501"},{"key":"e_1_2_7_41_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2006.12.001"},{"key":"e_1_2_7_42_1","volume-title":"MAP\u2010R for windows: multitrait \/ multi\u2010item analysis program \u2010 revised user's guide","author":"Ware JEJ","year":"1997"},{"key":"e_1_2_7_43_1","volume-title":"RUMM 2030 [computer program]","year":"1997"},{"key":"e_1_2_7_44_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF02293814"},{"key":"e_1_2_7_45_1","first-page":"95","article-title":"An index of person separation in latent trait theory, the traditional KR20 index, and the Guttman scale response pattern","volume":"9","author":"Andrich D","year":"1982","journal-title":"Educ Psychol Res"},{"key":"e_1_2_7_46_1","first-page":"33","volume-title":"Social methodology","author":"Andrich D","year":"1985"},{"key":"e_1_2_7_47_1","first-page":"59","volume-title":"Applications of latent trait and latent class models in the saocial sciences","author":"Andrich D","year":"1997"},{"key":"e_1_2_7_48_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0191-8869(03)00164-8"},{"key":"e_1_2_7_49_1","volume-title":"Rating scale analysis: Rasch measurement","author":"Wright BD","year":"1982"},{"key":"e_1_2_7_50_1","first-page":"200","article-title":"Formalising dimension and response violations of local independence in the unidimensional Rasch model","volume":"9","author":"Marais I","year":"2008","journal-title":"J Appl Meas"},{"key":"e_1_2_7_51_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF02310555"},{"key":"e_1_2_7_52_1","doi-asserted-by":"publisher","DOI":"10.1348\/014466506X96931"},{"key":"e_1_2_7_53_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0191-491X(02)00015-9"},{"key":"e_1_2_7_54_1","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S14399"},{"key":"e_1_2_7_55_1","doi-asserted-by":"publisher","DOI":"10.1067\/mse.2002.127096"},{"key":"e_1_2_7_56_1","doi-asserted-by":"publisher","DOI":"10.3102\/1076998611411913"},{"key":"e_1_2_7_57_1","doi-asserted-by":"publisher","DOI":"10.2466\/pms.1982.55.2.415"},{"key":"e_1_2_7_58_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.mlr.0000103528.48582.7c"},{"key":"e_1_2_7_59_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(07)60250-5"},{"key":"e_1_2_7_60_1","first-page":"307","article-title":"How accurate are lexile text measures?","volume":"7","author":"Stenner AJ","year":"2006","journal-title":"J Appl Meas"},{"key":"e_1_2_7_61_1","doi-asserted-by":"publisher","DOI":"10.1038\/219870a0"},{"key":"e_1_2_7_62_1","volume-title":"Assessing the Lexile Framework: results of a panel meeting NCES Working Paper Series, Working Paper No. 2001\u201308","author":"White S","year":"2001"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.23937","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.23937","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.23937","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,1,9]],"date-time":"2025-01-09T12:44:19Z","timestamp":1736426659000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.23937"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,7,26]]},"references-count":61,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2014,3]]}},"alternative-id":["10.1002\/mus.23937"],"URL":"https:\/\/doi.org\/10.1002\/mus.23937","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,7,26]]}}}