{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T02:52:52Z","timestamp":1790736772278,"version":"4.1.0"},"reference-count":27,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2022,5,11]],"date-time":"2022-05-11T00:00:00Z","timestamp":1652227200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004337","name":"Roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004337","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":[[2022,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Data regarding weight, height\/length, and growth status of patients with spinal muscular atrophy (SMA) who have received only supportive care are limited. This cross\u2010sectional study describes these measurements in patients with Type 1 and Types 2\/3 SMA and compares them with reference values from typically developing children.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Retrospective baseline data from three sites in the Pediatric Neuromuscular Clinical Research Network (Boston, New York, Philadelphia) were used. Descriptive statistics for weight, height\/length, body mass index\u2010for\u2010age, as well as weight\u2010for\u2010length and absolute and relative deviations from reference values (ie, 50th percentile from World Health Organization\/Centers for Disease Control growth charts) were calculated. Furthermore, growth status was reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 91 genetically confirmed patients with SMA receiving optimal supportive care and without any disease\u2010modifying treatment were stratified into Types 1 (<jats:italic>n<\/jats:italic>\u00a0=\u200928) and 2\/3 SMA (<jats:italic>n<\/jats:italic>\u00a0=\u200963). Patients with Type 1 SMA weighed significantly less (median\u00a0=\u00a0\u22127.5%) compared with reference values and patients with Types 2\/3 SMA were significantly shorter (mean\u00a0=\u00a0\u22123.0%) compared with reference values. The median weight was considerably below the 50th percentile in both groups of patients, even if they received a high standard of care and proactive feeding support.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>More research is needed to understand which factors influence growth longitudinally, and how to accurately capture growth in patients with SMA. Further research should investigate the best time to provide feeding support to avoid underweight, especially in patients with Type 1, and how to avoid the risk of overfeeding, especially in patients with Types 2\/3 SMA.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27556","type":"journal-article","created":{"date-parts":[[2022,4,6]],"date-time":"2022-04-06T12:46:53Z","timestamp":1649249213000},"page":"84-90","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United States"],"prefix":"10.1002","volume":"66","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1536-8145","authenticated-orcid":false,"given":"Basil T.","family":"Darras","sequence":"first","affiliation":[{"name":"Department of Neurology Boston Children's Hospital, Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0864-9296","authenticated-orcid":false,"given":"Sabrina","family":"Guye","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janine","family":"Hoffart","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Schneider","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0283-2065","authenticated-orcid":false,"given":"Isaac","family":"Gravestock","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ksenija","family":"Gorni","sequence":"additional","affiliation":[{"name":"PDMA, Neuroscience and Rare Disease, F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabine","family":"Fuerst\u2010Recktenwald","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renata S.","family":"Scalco","sequence":"additional","affiliation":[{"name":"Pharma Development Neurology, F. Hoffmann\u2010La Roche Ltd  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9351-7054","authenticated-orcid":false,"given":"Richard S.","family":"Finkel","sequence":"additional","affiliation":[{"name":"Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8420-0795","authenticated-orcid":false,"given":"Darryl C.","family":"De Vivo","sequence":"additional","affiliation":[{"name":"Departments of Neurology and Pediatrics Columbia University Irving Medical Center  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,5,11]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/4.4.631"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI58752"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-6-71"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24497"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.03.009"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.05.005"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190465"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073807305788"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2020.12.007"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.05.013"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2012.04.008"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_2_10_16_1","unstructured":"Healthy Weight Nutrition and Physical Activity.Centers for Disease Control and Prevention.https:\/\/www.cdc.gov\/healthyweight\/assessing\/bmi\/childrens_bmi\/about_childrens_bmi.html. Updated March 17 2021. Accessed March 4 2022."},{"key":"e_1_2_10_17_1","unstructured":"WHO Child Growth Standards: Length\/height\u2010for\u2010age weight\u2010for\u2010age weight\u2010for\u2010length weight\u2010for\u2010height and body mass index\u2010for\u2010age: Methods and development.https:\/\/www.who.int\/childgrowth\/standards\/technical_report\/en\/. Accessed March 4 2022."},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-02015-9"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clnu.2016.10.020"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2020.09.025"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"e_1_2_10_23_1","unstructured":"SPINRAZA\u00ae (nusinersen) US prescribing information.https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/209531lbl.pdf. Accessed March 4 2022."},{"key":"e_1_2_10_24_1","unstructured":"SPINRAZA\u00ae (nusinersen) EMA prescribing information.http:\/\/www.ema.europa.eu\/docs\/en_GB\/document_library\/EPAR_\u2010_Product_Information\/human\/004312\/WC500229704.pdf. Accessed March 4 2022."},{"key":"e_1_2_10_25_1","unstructured":"ZOLGENSMA\u00ae (onasemnogene abeparvovec\u2010xioi) US prescribing information.https:\/\/www.fda.gov\/media\/126109\/download. Accessed March 4 2022."},{"key":"e_1_2_10_26_1","unstructured":"ZOLGENSMA\u00ae (onasemnogene abeparvovec\u2010xioi) EMA prescribing information.https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/zolgensma-epar-product-information_en.pdf. Accessed March 4 2022."},{"key":"e_1_2_10_27_1","unstructured":"Evrysdi (risdiplam) EMA prescribing information.https:\/\/www.ema.europa.eu\/en\/documents\/product\u2010information\/evrysdi\u2010epar\u2010product\u2010information_en.pdf. Accessed March 4 2022."},{"key":"e_1_2_10_28_1","unstructured":"Evrysdi (risdiplam). US prescribing information.https:\/\/www.gene.com\/download\/pdf\/evrysdi_prescribing.pdf. Accessed March 4 2022."}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27556","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27556","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27556","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,24]],"date-time":"2023-08-24T12:34:32Z","timestamp":1692880472000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27556"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5,11]]},"references-count":27,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2022,7]]}},"alternative-id":["10.1002\/mus.27556"],"URL":"https:\/\/doi.org\/10.1002\/mus.27556","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,5,11]]},"assertion":[{"value":"2021-03-02","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-04-04","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-05-11","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}