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The efficacy in adults with long disease history and advanced disease status is still widely unknown; the same applies to specific expectations of adult SMA patients and to what extent they are met and may impact outcome measures.<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Methods<\/jats:title>\n<jats:p>In a longitudinal monocentric study in adult patients with SMA types 2\u20134, the Stanford Expectations of Treatment Scale (SETS) was assessed prior to and during nusinersen treatment. Treatment outcome was evaluated using patient-reported outcomes (PROs) as well as objectively quantifiable motor outcome measures.<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Results<\/jats:title>\n<jats:p>Adult SMA patients had high expectations of nusinersen\u00a0treatment effectiveness regarding increase in muscle strength and disease stabilization. Via PROs, 75% stated improvements in muscle strength, endurance and independence under therapy which was in line with slight improvements in quantifiable motor scores during a \u00a0ten month observation period. In contrast, patients only expressed few negative expectations which further decreased during therapy.<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Conclusions<\/jats:title>\n<jats:p>This study showed mainly positive treatment expectations and PROs in patients undergoing nusinersen treatment along with measurable functional improvement in adult SMA patients. Moreover, treatment expectations did not significantly influence outcome measures.<\/jats:p>\n<\/jats:sec>","DOI":"10.1007\/s00415-020-09847-8","type":"journal-article","created":{"date-parts":[[2020,5,2]],"date-time":"2020-05-02T12:02:40Z","timestamp":1588420960000},"page":"2398-2407","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":47,"title":["Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy"],"prefix":"10.1007","volume":"267","author":[{"given":"Alma","family":"Osmanovic","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gresa","family":"Ranxha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mareike","family":"Kumpe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lars","family":"M\u00fcschen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camilla","family":"Binz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flavia","family":"Wiehler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lejla","family":"Paracka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonja","family":"K\u00f6rner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katja","family":"Kollewe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanne","family":"Petri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivia","family":"Schreiber-Katz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,5,2]]},"reference":[{"issue":"4","key":"9847_CR1","doi-asserted-by":"publisher","first-page":"473","DOI":"10.1007\/s40265-017-0711-7","volume":"77","author":"SM Hoy","year":"2017","unstructured":"Hoy SM (2017) Nusinersen: first global approval. 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Australasia and Europe J Neurol 261(1):152\u2013163. https:\/\/doi.org\/10.1007\/s00415-013-7154-1","journal-title":"Australasia and Europe J Neurol"}],"container-title":["Journal of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-020-09847-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00415-020-09847-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-020-09847-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,5,3]],"date-time":"2021-05-03T16:41:53Z","timestamp":1620060113000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00415-020-09847-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,5,2]]},"references-count":32,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2020,8]]}},"alternative-id":["9847"],"URL":"https:\/\/doi.org\/10.1007\/s00415-020-09847-8","relation":{},"ISSN":["0340-5354","1432-1459"],"issn-type":[{"value":"0340-5354","type":"print"},{"value":"1432-1459","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,5,2]]},"assertion":[{"value":"19 December 2019","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"14 April 2020","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"17 April 2020","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"2 May 2020","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Compliance with ethical standards"}},{"value":"AO received honoraria from the Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.) and Biogen<b>.<\/b> GR and MK received travel funding from Biogen GmbH<b>.<\/b> LM, CB, FW and SK report no disclosures<b>.<\/b> LP received travel grants and\/or honoraria for lectures from Allergan, Ipsen, Merz. KK received travel grants and\/or honoraria for lectures from Allergan, Biogen, Ipsen, Lilly, Novartis, Merz, Teva. SP received honoraria from Biogen, Cytokinetics, Inc., Desitin Pharma, Novartis, Roche, Teva. OSK received scientific grants, travel grants and\/or honoraria from the German Neuromuscular Society (Deutsche Gesellschaft fuer Muskelkranke (DGM e.V.)), Novartis, Biogen, Jain Foundation.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"The study has been approved by the local ethics review board at Hannover Medical School and has, therefore, been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments. All patients gave their written informed consent before entering the study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}