{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T00:23:20Z","timestamp":1792887800631,"version":"4.5.0"},"reference-count":20,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T00:00:00Z","timestamp":1758585600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100030667","name":"Emory University School of Medicine - Department of Pediatrics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100030667","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":[[2025,12]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Identifying sensitive response measures is crucial for evaluating treatment efficacy in SMA patients. Current electrodiagnostic (EDX) measures include compound muscle action potential (CMAP), motor unit number index (MUNIX), and motor unit size index (MUSIX). Functional measures include Hammersmith functional motor scale extended (HFMSE), Children's Hospital of Philadelphia infant test of neuromuscular disorders (CHOP\u2010INTEND), and the revised upper limb module (RULM). Here, we compared EDX to traditional functional measures for monitoring treatment response in pediatric SMA.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A retrospective chart review of 16 non\u2010ambulatory, late\u2010onset (&gt;\u20096\u2009months of age) SMA children seen between January 2017 and June 2024 was performed. Motor nerve conduction study (NCS) recordings from the right median\u2010abductor pollicis brevis (APB) and ulnar\u2010abductor digiti minimi (ADM) nerve muscle pairs and functional scores were compared using Spearman correlation and a generalized linear mixed model (GLMM).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      EDX measures were significantly reduced in SMA children compared to healthy, age\u2010matched controls (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05). Median abductor pollicis brevis (APB) CMAP amplitudes demonstrated moderate to strong correlations with CHOP\u2010INTEND (\n                      <jats:italic>r<\/jats:italic>\n                      \u2009=\u20090.64,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05) and RULM (\n                      <jats:italic>r<\/jats:italic>\n                      \u2009=\u20090.77,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05) scores, and the GLMM revealed that APB CMAP significantly decreased over time (Beta\u2009=\u2009\u22120.02,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05) in the non\u2010treatment compared to the treatment group. HMFSE showed a declining trend in patients who received only risdiplam.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Median\u2010APB CMAP amplitude demonstrated sensitivity in capturing treatment response. A prospective study comparing nusinersen and risdiplam in a larger, clinically diverse cohort with a longer follow\u2010up period is needed.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70030","type":"journal-article","created":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T08:16:59Z","timestamp":1758615419000},"page":"1250-1258","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Electrodiagnostic and Functional Biomarkers for Non\u2010Ambulatory Children With Late\u2010Onset Spinal Muscular Atrophy"],"prefix":"10.1002","volume":"72","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4473-907X","authenticated-orcid":false,"given":"Mariana Minei","family":"Ogata","sequence":"first","affiliation":[{"name":"Universidad Peruana Cayetano Heredia  Lima Peru"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vishva","family":"Natarajan","sequence":"additional","affiliation":[{"name":"Geisel School of Medicine at Dartmouth  Hanover New Hampshire USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sriharsha","family":"Sripadrao","sequence":"additional","affiliation":[{"name":"Medical College of Georgia at Augusta University  Augusta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soham","family":"Verma","sequence":"additional","affiliation":[{"name":"Johns Creek High School  Johns Creek Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura M.","family":"Johnson","sequence":"additional","affiliation":[{"name":"Pediatric Biostatistics Core, Department of Pediatrics Emory University  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Durga","family":"Shah","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Ritchey","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberly","family":"Carvell","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1992-4556","authenticated-orcid":false,"given":"Sumit","family":"Verma","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology Emory University  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,9,23]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1177\/11772719211035643"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinph.2018.04.614"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2102047"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006502"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000007527"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1090\u20103798(03)00060\u20106"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25430"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21838"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27372"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2014.11.012"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26909"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1213\/ANE.0000000000002864"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26087"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinph.2016.06.011"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awac252"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp\u20102011\u2010301456"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinph.2018.11.009"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm13226881"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28470"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70030","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,10]],"date-time":"2025-11-10T17:12:58Z","timestamp":1762794778000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.70030"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9,23]]},"references-count":20,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2025,12]]}},"alternative-id":["10.1002\/mus.70030"],"URL":"https:\/\/doi.org\/10.1002\/mus.70030","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,9,23]]},"assertion":[{"value":"2025-02-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-09-12","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-09-23","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}