This longitudinal mixed methods study evaluated the perceived efficacy of salbutamol in adult patients with spinal muscular atrophy (SMA) and the usefulness of the World Health Organization Disability Assessment Schedule II (WHODAS II) and Fatigue Severity Scale (FSS) for its measurement. Ten patients were interviewed and completed WHODAS II and FSS questionnaires to assess disability and fatigue at 2 time-points. Inductive thematic analysis was used for qualitative data. The non-parametric Wilcoxon test was performed for quantitative analysis. All participants reported an improvement in their condition after salbutamol consumption. WHODAS II and FSS reliably captured changes in patients’ disability and fatigue.
