{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Osmanovic A"],"funding":["PRACTIS – Clinician Scientist Program of Hannover Medical School, funded by the German Research Foundation"],"pagination":["1756286421998902"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7940734"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["14"],"pubmed_abstract":["<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 treatme"],"journal":["Therapeutic advances in neurological disorders"],"pubmed_title":["Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy."],"pmcid":["PMC7940734"],"funding_grant_id":["ME 3696/3-1"],"pubmed_authors":["Ranxha G","Cordts I","Muschen LH","Kumpe M","Binz C","Hermann A","Petri S","Freigang M","Ludolph AC","Osmanovic A","Lingor P","Stolte B","Hagenacker T","Schreiber-Katz O","Wurster CD","Deschauer M","Gunther R"],"additional_accession":[]},"is_claimable":false,"name":"Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.","description":"<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 treatme","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021","modification":"2025-04-04T22:01:04.708Z","creation":"2025-04-04T22:01:04.708Z"},"accession":"S-EPMC7940734","cross_references":{"pubmed":["33747131"],"doi":["10.1177/1756286421998902"]}}