{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T08:43:04Z","timestamp":1785832984153,"version":"3.56.0"},"reference-count":33,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2021,6,16]],"date-time":"2021-06-16T00:00:00Z","timestamp":1623801600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2021,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background and purpose<\/jats:title><jats:p>This was an investigation of treatment expectations and of the perception of therapy in adult patients with 5q\u2010associated spinal muscular atrophy (5q\u2010SMA) receiving nusinersen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A prospective, non\u2010interventional observational study of nusinersen treatment in adult 5q\u2010SMA patients was conducted at nine SMA centers in Germany. The functional status, treatment expectations and perceived outcomes were assessed using the Amyotrophic Lateral Sclerosis Functional Rating Scale\u2014extended (ALS\u2010FRS\u2010ex), the Measure Yourself Medical Outcome Profile (MYMOP2), the Treatment Satisfaction Questionnaire for Medication (TSQM\u20109) and the Net Promoter Score (NPS).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In all, 151 patients were included with a median age of 36\u00a0years (15\u201369\u00a0years). SMA type 3 (<jats:italic>n<\/jats:italic>\u00a0=\u00a090, 59.6%) prevailed, followed by type 2 (33.8%) and type 1 (6.6%). In SMA types 1\u20133, median ALS\u2010FRS\u2010ex scores were 25, 33 and 46 (of 60 scale points), respectively. MYMOP2 identified distinct treatment expectations: head verticalization (<jats:italic>n<\/jats:italic>\u00a0=\u00a013), bulbar function (<jats:italic>n<\/jats:italic>\u00a0=\u00a016), arm function (<jats:italic>n<\/jats:italic>\u00a0=\u00a065), respiration (<jats:italic>n<\/jats:italic>\u00a0=\u00a015), trunk function (<jats:italic>n<\/jats:italic>\u00a0=\u00a034), leg function (<jats:italic>n<\/jats:italic>\u00a0=\u00a076) and generalized symptoms (<jats:italic>n<\/jats:italic>\u00a0=\u00a077). Median symptom severity decreased during nusinersen treatment (median observational period 6.1\u00a0months, 0.5\u201316\u00a0months) from 3.7 to 3.3 MYMOP2 score points (<jats:italic>p\u00a0<\/jats:italic>&lt;\u00a00.001). The convenience of drug administration was critical (49.7 of 100 TSQM\u20109 points, SD 22); however, the overall treatment satisfaction was high (74.3, SD 18) and the recommendation rating very positive (NPS +66).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Nusinersen was administered across a broad range of ages, disease durations and motor function deficits. Treatment expectations were highly differentiated and related to SMA type and functional status. Patient\u2010reported outcomes demonstrated a positive perception of nusinersen therapy in adult patients with 5q\u2010SMA.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ene.14902","type":"journal-article","created":{"date-parts":[[2021,5,8]],"date-time":"2021-05-08T17:54:02Z","timestamp":1620496442000},"page":"2582-2595","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":30,"title":["Treatment expectations and perception of therapy in adult patients with spinal muscular atrophy receiving nusinersen"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2736-7350","authenticated-orcid":false,"given":"Thomas","family":"Meyer","sequence":"first","affiliation":[{"name":"Department of Neurology Center for ALS, SMA and other Motor Neuron Disorders Charit\u00e9\u2014Universit\u00e4tsmedizin Berlin Berlin Germany"},{"name":"Ambulanzpartner Soziotechnologie APST GmbH Berlin 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