Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.

ABSTRACT:

Background

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.

Methods

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

SUBMITTER: Osmanovic A 

PROVIDER: S-EPMC7940734 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature