{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T11:21:03Z","timestamp":1788520863691,"version":"build-2803163510"},"reference-count":60,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2026,1,21]],"date-time":"2026-01-21T00:00:00Z","timestamp":1768953600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,1,21]],"date-time":"2026-01-21T00:00:00Z","timestamp":1768953600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","award":["NG2-170044"],"award-info":[{"award-number":["NG2-170044"]}],"id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","award":["OR2-189333"],"award-info":[{"award-number":["OR2-189333"]}],"id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Clinical Outcome Assessments (COAs) are essential for monitoring progression and treatment response in neuromuscular diseases. However, substantial variability exists in training, confidence, and implementation of COAs among clinical evaluators working with individuals with Spinal Muscular Atrophy (SMA). This study aimed to identify and address these gaps within the Canadian clinical context through a phased educational initiative guided by the Rare Knowledge Mining Methodological Framework (RKMMF).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A qualitative, phased design was used. A needs assessment with 71 physiotherapists and occupational therapists via focus groups identified inconsistent access to SMA-specific training and challenges in applying standardized assessments. Based on these findings, expert faculty co-developed and delivered bilingual workshops incorporating real-world evaluation videos, simulation-based learning, and multidisciplinary case discussions. Pre- and post-workshop surveys, based on an adapted Kirkpatrick Model, measured changes in familiarity, preparedness, and clinical confidence. Data were analyzed using thematic content analysis and descriptive statistics.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Seventy-nine evaluators from Canada participated. Pre-workshop data revealed major gaps in familiarity with SMA-specific COAs. Post-workshop surveys indicated a 75% average increase in self-reported preparedness, with the greatest gains in the Adapted Test of Neuromuscular Disorders 3.0. Four key themes emerged: limited training and support networks; the critical role of multidisciplinary collaboration; constraints of current COAs due to ceiling and floor effects; and the value of integrating patient-reported outcomes in clinical practice.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Peer-led, evidence-informed workshops significantly improved clinical preparedness in SMA assessment. These findings support the need for ongoing training strategies and demonstrate the RKMMF as a scalable approach for capacity-building in rare disease care.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/22143602261416298","type":"journal-article","created":{"date-parts":[[2026,1,21]],"date-time":"2026-01-21T19:38:11Z","timestamp":1769024291000},"page":"1014-1026","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Strengthening clinical capacity in spinal muscular atrophy: Developing and implementing training on clinical outcome assessments"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-4225-9075","authenticated-orcid":false,"given":"Homira","family":"Osman","sequence":"first","affiliation":[{"name":"Muscular Dystrophy Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-3334-8763","authenticated-orcid":false,"given":"Maria","family":"Masnata","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-9728-3790","authenticated-orcid":false,"given":"Zainab","family":"Adamji","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xavier","family":"Rodrigue","sequence":"additional","affiliation":[{"name":"Institut de r\u00e9adaptation en d\u00e9ficience physique de Qu\u00e9bec"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cam-Tu \u00c9milie","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire Sainte-Justine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1048-266X","authenticated-orcid":false,"given":"Jeremy","family":"Slayter","sequence":"additional","affiliation":[{"name":"Dalhousie University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"Beattie","sequence":"additional","affiliation":[{"name":"Children's Hospital of Eastern Ontario Research Institute, Ottawa, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stacey","family":"Lintern","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanns","family":"Lochmuller","sequence":"additional","affiliation":[{"name":"Children's Hospital of Eastern Ontario Research Institute, Ottawa, Canada"},{"name":"The Ottawa Hospital, Ottawa, Canada"},{"name":"University of Ottawa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colleen","family":"O\u2019Connell","sequence":"additional","affiliation":[{"name":"Dalhousie University"},{"name":"Stan Cassidy Centre for Rehabilitation"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cynthia","family":"Gagnon","sequence":"additional","affiliation":[{"name":"Universit\u00e9 de Shebrooke, Sherbrooke, Canada"},{"name":"Centre de recherche et d\u2019innovation du Centre int\u00e9gr\u00e9e universitaire du Saguenay-Lac-St-Jean, Montreal, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0187-2199","authenticated-orcid":false,"given":"Jodi","family":"Warman-Chardon","sequence":"additional","affiliation":[{"name":"The Ottawa Hospital, Ottawa, Canada"},{"name":"University of Ottawa"},{"name":"Department of Genetics, Children's Hospital of Eastern Ontario\/Research Institute, Ottawa, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,1,21]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190424"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamapediatrics.2024.0492"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000209151"},{"key":"e_1_3_3_5_2","unstructured":"Clinical Outcome Assessment (COA): Frequently Asked Questions | FDA https:\/\/www.fda.gov\/about-fda\/clinical-outcome-assessment-coa-frequently-asked-questions#COADefinition (accessed 11 June 2025)."},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2017.08.2051"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-6-71"},{"key":"e_1_3_3_9_2","first-page":"1","article-title":"Spinal muscular atrophy","author":"Prior TW","year":"2024","unstructured":"Prior TW, Leach ME, Finanger EL. 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