An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy.

ABSTRACT:

Background

Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SMA under nusinersen.

Methods

We recorded forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and peak expiratory flow (PEF) during nusinersen treatment in 38 adult SMA patients. Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 36-Item Short Form Health Survey (SF-36) questionnaire and Fatigue Severity Scale (FSS) were recorded and correlations between muscle function, QoL, fatigue and respiratory parameters were ana

SUBMITTER: Bjelica B 

PROVIDER: S-EPMC10106012 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature