biostudies-literatureOsmanovic APRACTIS – Clinician Scientist Program of Hannover Medical School, funded by the German Research Foundation1756286421998902https://www.ebi.ac.uk/biostudies/studies/S-EPMC7940734biostudies-literatureUnknown14<h4>Background</h4>Nusinersen was the first approved disease-modifying therapy for all 5q-spinal muscular atrophy (SMA) patients regardless of age or disease severity. Its efficacy in adults has recently been demonstrated in a large cohort by motor outcome measures, which were only partially suitable to detect changes in very mildly or severely affected patients. Patient-reported outcome measures (PROs) have been suggested as a valuable addition. Here, we aimed to assess treatment satisfaction and investigate whether it may be a useful PRO to monitor SMA patients.<h4>Methods</h4>We enrolled 91 mainly adult 5q-SMA patients treated with nusinersen in a national, multicenter, cross-sectional observational study. 21 patients underwent longitudinal follow up. Patients' satisfaction with treatmeTherapeutic advances in neurological disordersTreatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.PMC7940734ME 3696/3-1Ranxha GCordts IMuschen LHKumpe MBinz CHermann APetri SFreigang MLudolph ACOsmanovic ALingor PStolte BHagenacker TSchreiber-Katz OWurster CDDeschauer MGunther RfalseTreatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.<h4>Background</h4>Nusinersen was the first approved disease-modifying therapy for all 5q-spinal muscular atrophy (SMA) patients regardless of age or disease severity. Its efficacy in adults has recently been demonstrated in a large cohort by motor outcome measures, which were only partially suitable to detect changes in very mildly or severely affected patients. Patient-reported outcome measures (PROs) have been suggested as a valuable addition. Here, we aimed to assess treatment satisfaction and investigate whether it may be a useful PRO to monitor SMA patients.<h4>Methods</h4>We enrolled 91 mainly adult 5q-SMA patients treated with nusinersen in a national, multicenter, cross-sectional observational study. 21 patients underwent longitudinal follow up. Patients' satisfaction with treatme2021-01-01T00:00:00Z20212025-04-04T22:01:04.708Z2025-04-04T22:01:04.708ZS-EPMC79407343374713110.1177/1756286421998902