{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T00:23:18Z","timestamp":1792887798650,"version":"4.5.0"},"reference-count":17,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2026,2,2]],"date-time":"2026-02-02T00:00:00Z","timestamp":1769990400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,2,2]],"date-time":"2026-02-02T00:00:00Z","timestamp":1769990400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100001807","name":"Funda\u00e7\u00e3o de Amparo \u00e0 Pesquisa do Estado de S\u00e3o Paulo","doi-asserted-by":"publisher","award":["2021\/06739\u20104"],"award-info":[{"award-number":["2021\/06739\u20104"]}],"id":[{"id":"10.13039\/501100001807","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,4]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Natural history data for adult patients with spinal muscular atrophy (SMA) remain scarce, which is particularly relevant in the current therapeutic era. This study aimed to identify the most sensitive clinical, patient\u2010reported, and neurophysiological measures to detect short\u2010term disease progression in untreated adult SMA patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This prospective, one\u2010year longitudinal study included 21 genetically confirmed adult patients with SMA types 2B and 3. Clinician\u2010reported outcomes (CROs) included the Motor Function Measure (MFM), Hammersmith Functional Motor Scale Expanded (HFMSE), and Revised Upper Limb Module (RULM). Additionally, patient\u2010reported outcomes (PROs) were assessed using the Modified Fatigue Impact Scale (MFIS). Neurophysiological evaluations included compound muscle action potential (CMAP) amplitude and motor unit number index (MUNIX) recorded from the ulnar nerve. Sensitivity to change was determined using standardized response means (SRMs), and associations between clinical and neurophysiological data were analyzed via Spearman correlation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      The majority of participants were non\u2010ambulatory (16\/21). The MFM total score was the only outcome to show a statistically significant decline over 12\u2009months (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.02), with the highest SRM (\u22120.55), indicating superior sensitivity. MFM also demonstrated the strongest correlations with CMAP amplitude (\n                      <jats:italic>\u03c1<\/jats:italic>\n                      \u2009=\u20090.90) and MUNIX (\n                      <jats:italic>\u03c1<\/jats:italic>\n                      \u2009=\u20090.75), compared to other CROs. No significant longitudinal changes were observed in RULM, HFMSE, MFIS, CMAP, or MUNIX.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Among evaluated outcome measures, the MFM was the most sensitive to short\u2010term progression and most closely aligned with neurophysiological markers. These findings support the use of MFM as a primary outcome in clinical trials involving adult SMA patients.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70169","type":"journal-article","created":{"date-parts":[[2026,2,2]],"date-time":"2026-02-02T11:10:16Z","timestamp":1770030616000},"page":"645-649","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["How to Best Measure Disease Progression in Adult Spinal Muscular Atrophy Patients: A Clinical and Neurophysiological Study"],"prefix":"10.1002","volume":"73","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8318-9766","authenticated-orcid":false,"given":"Felipe Franco","family":"da Gra\u00e7a","sequence":"first","affiliation":[{"name":"Department of Neurology, School of Medical Sciences University of Campinas (UNICAMP)  Campinas S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Iwabe","sequence":"additional","affiliation":[{"name":"Department of Neurology, School of Medical Sciences University of Campinas (UNICAMP)  Campinas S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anamarli","family":"Nucci","sequence":"additional","affiliation":[{"name":"Department of Neurology, School of Medical Sciences University of Campinas (UNICAMP)  Campinas S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thiago Junqueira Ribeiro","family":"de Rezende","sequence":"additional","affiliation":[{"name":"Department of Neurology, School of Medical Sciences University of Campinas (UNICAMP)  Campinas S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0898-2419","authenticated-orcid":false,"suffix":"Jr","given":"Marcondes Cavalcante","family":"Fran\u00e7a","sequence":"additional","affiliation":[{"name":"Department of Neurology, School of Medical Sciences University of Campinas (UNICAMP)  Campinas S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,2,2]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572\u2010022\u201000380\u20108"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(23)00419\u20102"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND\u2010221595"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1177\/2329048X211008725"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2005.03.004"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25430"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073811420294"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neulet.2020.135488"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2021.02.019"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1590\/S1413\u201035552008000500012"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1590\/s0004\u2010282x2007000400024"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1109\/TBME.2004.834281"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2021.770423"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16309"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1213\/ANE.0000000000002864"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16517"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51281"}],"container-title":["Muscle &amp; 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