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The aim of this study was to assess the construct validity and responsiveness of several outcome measures in adult SMA patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients older than 15\u2009years and followed up in five referral centres for at least 6\u00a0months, between October 2015 and August 2020, with a motor function scale score (Hammersmith Functional Motor Scale Expanded [HFMSE], Revised Upper Limb module [RULM]) were included. Bedside functional scales (Egen Klassification [EK2], Revised Amyotrophic Lateral Sclerosis Functional Rating Scale [ALSFRS\u2010R]) were also collected when available. Spearman's rho correlations (rs) and Bangdiwala's concordance test (B) were used to evaluate the scales' construct validity. Monthly slopes of change were used to calculate their responsiveness of the scales.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The study included 79 SMA patients, followed up for a mean of 16\u2009months. All scales showed strong correlations with each other (rs\u2009&gt;\u20090.70). A floor effect in motor function scales was found in the weakest patients (HFMSE\u2009&lt;\u20095 and RULM\u2009&lt;\u200910), and a ceiling effect was found in stronger patients (HFMSE\u2009&gt;\u200960 and RULM\u2009&gt;\u200935). The ALSFRS\u2010R (B\u00a0=\u00a00.72) showed a strong ability to discriminate between walkers, sitters and non\u2010sitters, and the HFMSE (B\u00a0=\u00a00.86) between walkers and sitters. The responsiveness was low overall, although in treated patients a moderate responsiveness was found for the ALSFRS\u2010R and HFMSE in walkers (0.69 and 0.61, respectively) and for EK2 in sitters (0.65) and non\u2010sitters (0.60).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This study shows the validity and limitations of the scales most frequently used to assess adult SMA patients. Overall, bedside functional scales showed some advantages over motor scales, although all showed limited responsiveness.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.15542","type":"journal-article","created":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T09:59:20Z","timestamp":1662026360000},"page":"3666-3675","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":40,"title":["Validation of motor and functional scales for the evaluation of adult patients with 5q spinal muscular atrophy"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3043-7938","authenticated-orcid":false,"given":"Juan F.","family":"V\u00e1zquez\u2010Costa","sequence":"first","affiliation":[{"name":"Neuromuscular Unit, Department of Neurology IIS La Fe, Hospital Universitario y Polit\u00e9cnico la Fe  Valencia Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER)  Valencia Spain"},{"name":"Department of Medicine Universitat de Val\u00e8ncia  Valencia Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00f3nica","family":"Povedano","sequence":"additional","affiliation":[{"name":"Motor Neuron Unit, Neurology Department Bellvitge Hospital\u2010IDIBELL  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9s E.","family":"Nascimiento\u2010Osorio","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Neuropediatric Department Institut de Recerca Pedi\u00e0trica Hospital Sant Joan de D\u00e9u  Barcelona Spain"},{"name":"Center for the Biomedical Research on Rare Diseases (CIBERER), ISCIII  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Moreno Escribano","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Neurology Department Hospital Cl\u00ednico Universitario Virgen de la Arrixaca  Murcia 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