{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T01:25:11Z","timestamp":1792805111241,"version":"4.5.0"},"reference-count":38,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,5,11]],"date-time":"2026-05-11T00:00:00Z","timestamp":1778457600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Few studies have investigated nutrition as a primary outcome of disease modifying therapy (DMT) in spinal muscular atrophy (SMA). This study aimed to describe nutrition outcomes of DMT in children with SMA 1 and 2.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Children \u2264\u200918\u2009years old with SMA 1 or 2 treated with DMTs, and untreated children with SMA 1 were included. Retrospective data (anthropometry, feeding, dietetic intervention) were collected from medical records for their duration of care in a tertiary pediatric neuromuscular clinic. Data were analyzed descriptively and reported as median (interquartile range) or percentages.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Seventeen treated and 19 untreated children with SMA 1 and 23 treated children with SMA 2 were included. Age (months) at DMT initiation was 3.8 (1.8, 8.7) and 60.3 (28.5, 127.5) for children with SMA 1 and 2 respectively. SMA 1: At 6\u2009months old, weight\n                      <jats:italic>z<\/jats:italic>\n                      \u2010score was \u22120.25 (\u22121.21, 0.07,\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20097) for treated and \u22122.67 (\u22123.09, 0.13,\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20097) for untreated children. Tube feeding was initiated in 82% treated and 89% untreated children; time to initiation (months) was 16.2 for treated children and 6.6 for untreated children. Three treated children ceased tube feeding after 0.6\u201319\u2009months. SMA 2: Nutrition outcomes were described over 4\u2009years of DMT. All were orally fed. Minimal changes were observed in weight\n                      <jats:italic>z<\/jats:italic>\n                      \u2010score trajectories or feeding.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Nutrition outcomes were improved in treated children with SMA 1 and stable in treated children with SMA 2. Regular dietetic assessment and intervention remain integral to SMA care. Prospective studies are required.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70275","type":"journal-article","created":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T02:14:26Z","timestamp":1778552066000},"page":"198-206","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["A Retrospective Cohort Study of Nutrition Outcomes in Children With Spinal Muscular Atrophy Type 1 and 2 Treated With Disease Modifying Therapies"],"prefix":"10.1002","volume":"74","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7886-551X","authenticated-orcid":false,"given":"Katie","family":"O'Brien","sequence":"first","affiliation":[{"name":"Department of Nutrition, Dietetics and Food Monash University, Faculty of Medicine Nursing and Health Sciences  Melbourne Victoria Australia"},{"name":"Department of Nutrition and Food Services Royal Children's Hospital  Melbourne Victoria Australia"},{"name":"Neurosciences Research, Murdoch Children's Research Institute  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kay","family":"Nguo","sequence":"additional","affiliation":[{"name":"Department of Nutrition, Dietetics and Food Monash University, Faculty of Medicine Nursing and Health Sciences  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6704-8402","authenticated-orcid":false,"given":"Eppie M.","family":"Yiu","sequence":"additional","affiliation":[{"name":"Neurosciences Research, Murdoch Children's Research Institute  Melbourne Victoria Australia"},{"name":"Department of Neurology Royal Children's Hospital  Melbourne Victoria Australia"},{"name":"Department of Paediatrics Faculty of Medicine Dentistry and Health Sciences, University of Melbourne  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian R.","family":"Woodcock","sequence":"additional","affiliation":[{"name":"Neurosciences Research, Murdoch Children's Research Institute  Melbourne Victoria Australia"},{"name":"Department of Neurology Royal Children's Hospital  Melbourne Victoria Australia"},{"name":"Department of Paediatrics Faculty of Medicine Dentistry and Health Sciences, University of Melbourne  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8566-9341","authenticated-orcid":false,"given":"Natassja","family":"Billich","sequence":"additional","affiliation":[{"name":"Molecular Therapies Research, Murdoch Children's Research Institute  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zoe E.","family":"Davidson","sequence":"additional","affiliation":[{"name":"Department of Nutrition, Dietetics and Food Monash University, Faculty of Medicine Nursing and Health Sciences  Melbourne Victoria Australia"},{"name":"Neurosciences Research, Murdoch Children's Research Institute  Melbourne Victoria Australia"},{"name":"Department of Neurology Royal Children's Hospital  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,5,11]]},"reference":[{"key":"e_1_2_12_2_1","unstructured":"\u201cFDA Approves First Drug for Spinal Muscular Atrophy \u201dU.S. Food and Drug Administration Website updated 28 March 2018 accessed April 15 2025 https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010approves\u2010first\u2010drug\u2010spinal\u2010muscular\u2010atrophy."},{"key":"e_1_2_12_3_1","unstructured":"\u201cFDA Approves Innovative Gene Therapy to Treat Pediatric Patients With Spinal Muscular Atrophy A Rare Disease and Leading Genetic Cause of Infant Mortality \u201dU.S. Food and Drug Administration Website accessed 15 April 2025 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