Goal Attainment Scale light captures clinically meaningful changes in Adolescents and Adults with Spinal Muscular Atrophy Treated with Risdiplam

The study evaluated the usefulness of the Goal Attainment Scale light (GAS light) for assessing response to risdiplam treatment in patients with spinal muscular atrophy (SMA) aged 15 years and older. The study included 44 patients (median age 32 years, 56.8% female), of which 31.8% were non-sitters, 56.8% were sitters, and 11.4% were walkers. Patients were examined before treatment initiation and at 12 and 24 months after starting risdiplam using various motor function and functional ability tests. After 24 months of treatment, GAS light detected significant improvements across all patient groups, whereas standard tests showed improvements only in certain groups. The minimal clinically important change for GAS light was 6.5 points, and 64.5% of patients achieved clinically meaningful improvement in their goals. GAS light proved to be a suitable and sensitive patient-centered tool that can complement standard measurement methods when evaluating treatment effectiveness in adults with SMA.