{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,4]],"date-time":"2026-11-04T20:37:14Z","timestamp":1793824634467,"version":"4.6.1"},"reference-count":11,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2025,6,12]],"date-time":"2025-06-12T00:00:00Z","timestamp":1749686400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100011724","name":"Muscular Dystrophy UK","doi-asserted-by":"publisher","award":["07DN02; 37787"],"award-info":[{"award-number":["07DN02; 37787"]}],"id":[{"id":"10.13039\/100011724","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005614","name":"Biogen","doi-asserted-by":"publisher","award":["REC reference: 13\/LO\/1748, IRAS project ID: 122521"],"award-info":[{"award-number":["REC reference: 13\/LO\/1748, IRAS project ID: 122521"]}],"id":[{"id":"10.13039\/100005614","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100019256","name":"NIHR Great Ormond Street Hospital Biomedical Research Centre","doi-asserted-by":"publisher","award":["515048"],"award-info":[{"award-number":["515048"]}],"id":[{"id":"10.13039\/501100019256","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To describe bulbar function trajectories in patients with spinal muscular atrophy (SMA) type 1 treated with nusinersen in the UK and Italy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Method<\/jats:title>\n                    <jats:p>In two previously reported, retrospective, observational cohort studies, we observed the 2\u2010year change in the Children's Eating and Drinking Ability Scale (CEDAS) (the revised and optimized version of the Paediatric Functional Oral Intake Scale [p\u2010FOIS]) and Oral and Swallowing Ability Tool (OrSAT) in 44 patients treated on average at 8.3\u2009months (interquartile range\u2009=\u20094.1\u201314.4\u2009months), with data collected every 6\u2009months from treatment initiation. [Correction added on 30 June 2025 after first online publication: In the preceding sentence, 11.2 months (interquartile range = 4.1\u201324.7 months) has been changed to 8.3 months (interquartile range = 4.1\u201314.4 months).]<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The Italian cohort had more participants in the 1b group (symptom onset\u2009&gt;2\u2009weeks and\u2009&lt;3\u2009months), while the UK had more participants in the 1c group (symptom onset\u2009&lt;6\u2009months). Over 2\u2009years, the p\u2010FOIS\/CEDAS captured lack of bulbar improvement in the 1b group, with 40% displaying stability and 45% showing decline; in the 1c group, stability was captured (71%). OrSAT captured improvement in 47% of the 1b group and 43% of the 1c group at 2\u2009years; this was predominantly because of age\u2010related speech acquisition and feeding viscosities, where the item was not age\u2010appropriate at baseline.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>The p\u2010FOIS\/CEDAS and OrSAT measures capture complementary information on the effect of disease\u2010modifying treatments (DMTs) on bulbar function. Further studies are required to understand bulbar function trajectories in symptomatic and presymptomatic cohorts with SMA receiving different DMTs.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/dmcn.16387","type":"journal-article","created":{"date-parts":[[2025,6,12]],"date-time":"2025-06-12T12:48:33Z","timestamp":1749732513000},"page":"1590-1600","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen"],"prefix":"10.1111","volume":"67","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2795-9898","authenticated-orcid":false,"given":"Georgia","family":"Stimpson","sequence":"first","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lavinia","family":"Fanelli","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit Catholic University  Rome Italy"},{"name":"Centro Clinico NeMO Fondazione Policlinico Universitario Agostino Gemelli Istituto di Ricovero e Cura a Carattere Scientifico  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eleanor","family":"Conway","sequence":"additional","affiliation":[{"name":"National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"Johnson","sequence":"additional","affiliation":[{"name":"Speech and Language Therapy Department Great Ormond Street Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Berti","sequence":"additional","affiliation":[{"name":"Centro Clinico NeMO Fondazione Policlinico Universitario Agostino Gemelli Istituto di Ricovero e Cura a Carattere Scientifico  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariacristina","family":"Scoto","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health  London UK"},{"name":"Neuromuscular Department Great Ormond Street Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health  London UK"},{"name":"National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Unit Catholic University  Rome Italy"},{"name":"Centro Clinico NeMO Fondazione Policlinico Universitario Agostino Gemelli Istituto di Ricovero e Cura a Carattere Scientifico  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4871-6692","authenticated-orcid":false,"given":"Giovanni","family":"Baranello","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health  London UK"},{"name":"National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the p\u2010FOIS\/CEDAS and OrSAT Working Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,6,12]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2017.189"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"e_1_2_9_4_1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3389\/fped.2020.00004","article-title":"Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports","volume":"8","author":"Hully M","year":"2020","journal-title":"Front Pediatr."},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000796"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15542"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15171"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1136\/archdischild-2022-323899"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200614"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2024.02.003"}],"container-title":["Developmental Medicine &amp; 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