{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T06:49:51Z","timestamp":1784616591751,"version":"3.55.0"},"reference-count":21,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2021,4,3]],"date-time":"2021-04-03T00:00:00Z","timestamp":1617408000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2021,7,30]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Spinal muscular atrophy (SMA) is a rare genetic disease characterized by progressive muscular weakness and atrophy resulting from motor neuron degeneration. Limited information is available on disease progression among older SMA patients, particularly adults.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>This study sought to characterize the natural history of SMA among adult patients in US hospital settings through the assessment of symptoms, complications, costs, and healthcare resource utilization (HRU) over 3 years before the availability of disease-modifying therapies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>The study population included adult (\u226518 years) patients with inpatient and\/or hospital-based outpatient discharge records and \u22652 primary or secondary SMA ICD-9 codes \u226530 days apart in the Premier Healthcare Database during the main study period (2007\u20132014). Index date was the date of the first SMA ICD-9 code. The frequency of SMA-related symptoms and complications was assessed 1 year preindex through 2 years postindex to characterize disease progression. Costs and HRU were also assessed across the study period.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>A total of 446 adult patients from 337 US hospitals met inclusion criteria for these analyses. All evaluated SMA-related symptoms and complications increased steadily over time, from 1 year preindex to 2 years postindex both overall and in each age group. Adult patients with SMA had increasing total costs and HRU over the 3-year study period: total costs were $1,759 preindex and $12,308 by 2 years postindex.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Findings are consistent with increasing disease burden over time and support the progressive nature of SMA for adult patients with hospital interactions.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-200624","type":"journal-article","created":{"date-parts":[[2021,4,6]],"date-time":"2021-04-06T13:05:03Z","timestamp":1617714303000},"page":"569-578","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":4,"title":["Characterization of Adult Patients With SMA Treated in US Hospital Settings: A Natural History Study in the Premier Healthcare Database"],"prefix":"10.1177","volume":"8","author":[{"given":"Nicole B.","family":"Johnson","sequence":"first","affiliation":[{"name":"Biogen, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Crystal","family":"Proud","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of The King\u2019s Daughters, Norfolk, VA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina L.","family":"Wassel","sequence":"additional","affiliation":[{"name":"Premier Inc., Charlotte, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jill","family":"Dreyfus","sequence":"additional","affiliation":[{"name":"Premier Inc., Charlotte, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thos","family":"Cochrane","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela D.","family":"Paradis","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2021,4,3]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60921-6"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.7.1177"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1086\/338627"},{"key":"ref006","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0671-8"},{"key":"ref007","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0724-z"},{"key":"ref008","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.04.009"},{"key":"ref009","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.1995.00540290108025"},{"key":"ref010","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006241"},{"key":"ref011","unstructured":"Premier Applied Sciences\u00ae tRDoPI. Premier Healthcare Database white paper: Data that informs and performs. Available from: https:\/\/products.premierinc.com\/downloads\/PremierHealthcareDatabaseWhitepaper.pdf November 30, 2020]."},{"key":"ref012","doi-asserted-by":"publisher","DOI":"10.1007\/s41669-018-0093-0"},{"key":"ref013","doi-asserted-by":"publisher","DOI":"10.1080\/13696998.2019.1646263"},{"key":"ref014","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2018.11.002"},{"key":"ref015","unstructured":"The Lewin Group. Cost of amyotrophic lateral sclerosis, muscular dystrophy, and spinal muscular atrophy in the United States. https:\/\/www.mda.org\/sites\/default\/files\/Cost_Illness_Report_0.pdf. Accessed October 27, 2020 2012"},{"key":"ref016","doi-asserted-by":"publisher","DOI":"10.1177\/0194599814549156"},{"key":"ref017","doi-asserted-by":"publisher","DOI":"10.1093\/ije\/16.4.537"},{"key":"ref018","doi-asserted-by":"publisher","DOI":"10.1097\/BRS.0b013e3181e9f120"},{"key":"ref019","doi-asserted-by":"publisher","DOI":"10.1007\/s00586-015-4215-4"},{"key":"ref020","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc1511751"},{"issue":"9","key":"ref021","first-page":"1","volume":"68","author":"Kochanek KD","year":"2019","journal-title":"Natl Vital Stat Rep"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-200624","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.3233\/JND-200624","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-200624","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T23:49:41Z","timestamp":1777506581000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.3233\/JND-200624"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,4,3]]},"references-count":21,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2021,7,30]]}},"alternative-id":["10.3233\/JND-200624"],"URL":"https:\/\/doi.org\/10.3233\/jnd-200624","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,4,3]]}}}