{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T03:43:50Z","timestamp":1784778230647,"version":"3.55.0"},"reference-count":38,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2023,9,21]],"date-time":"2023-09-21T00:00:00Z","timestamp":1695254400000},"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":[[2023,11,7]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Limited qualitative data exist on the symptoms and impacts of spinal muscular atrophy (SMA) experienced by ambulant individuals. An ambulant module of the SMA Independence Scale (SMAIS) was developed to quantify the assistance required to perform everyday mobility-related activities.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>The objective of this study was to develop a patient-centered module that provides key insights into what constitutes independence for ambulant and near-ambulant individuals with SMA.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A stepwise, mixed-method approach was used. Semi-structured interviews were conducted in three waves with individuals with SMA and caregivers of children with SMA who were ambulant or near-ambulant (can walk \u22655 steps with support). Wave 1 interviews ( n\u200a=\u200a20) focused on concept elicitation. Wave 2 and 3 interviews ( n\u200a=\u200a15, both) involved completion and cognitive debriefing of items generated based on Wave 1 interviews. Therapeutic area experts were consulted throughout all key steps of the study. In particular, feedback was provided for item refinement and response option decisions. A macro-level preliminary, exploratory analysis, using Rasch Measurement Theory (RMT), provided insight on measurement properties.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Wave 1 resulted in 42 mobility and 11 instrumental activity of daily living (iADL) items. During Wave 2, participants defined independence as completing a task with supportive aids but without help from another person, leading to item refinement and modifications to the response scale. Lack of conceptual relevance and ceiling effects led to the removal of all iADL items after Wave 2, and 41 mobility items were tested in Wave 3. Final exploratory RMT and item refinement to reduce overlap led to a 27-item set related to mobility tasks.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Our study provides preliminary support for using the 27-item SMAIS\u2013Ambulatory Module for ambulant or near-ambulant individuals with SMA. Larger-scale analyses to further assess the psychometric properties of the scale are warranted.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-230096","type":"journal-article","created":{"date-parts":[[2023,9,22]],"date-time":"2023-09-22T12:18:47Z","timestamp":1695385127000},"page":"1093-1109","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":8,"title":["A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale"],"prefix":"10.1177","volume":"10","author":[{"given":"Hannah","family":"Staunton","sequence":"first","affiliation":[{"name":"Roche Products Ltd, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, AL7 1TW, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Cleanthous","sequence":"additional","affiliation":[{"name":"Modus Outcomes, 4th Floor St. James House, St. James Square, Cheltenham, GL50 3PR, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vanda","family":"Teodoro","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louise","family":"Barrett","sequence":"additional","affiliation":[{"name":"Modus Outcomes, 4th Floor St. James House, St. James Square, Cheltenham, GL50 3PR, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Braid","sequence":"additional","affiliation":[{"name":"Roche Products Ltd, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, AL7 1TW, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bethany","family":"Ewens","sequence":"additional","affiliation":[{"name":"Modus Outcomes, 4th Floor St. James House, St. James Square, Cheltenham, GL50 3PR, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"Cano","sequence":"additional","affiliation":[{"name":"Modus Outcomes, 4th Floor St. James House, St. James Square, Cheltenham, GL50 3PR, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanni","family":"Baranello","sequence":"additional","affiliation":[{"name":"The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, WC1N 3JH, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janbernd","family":"Kirschner","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics and Muscle Disorders, University of Freiburg, Faculty of Medicine, 79106, Freiburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa","family":"Belter","sequence":"additional","affiliation":[{"name":"Cure SMA, Elk Grove Village, IL, 60007, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Mayhew","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle Upon Tyne, NE1 3BZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,9,21]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2015.07.004"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"ref005","unstructured":"PriorTW, LeachME, FinangerE. 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