Exploring Treatment Expectations and Clinical Meaningfulness in Spinal Muscular Atrophy Using the Goal Attainment Scale.

This was an exploratory pilot at a single centre with no comparison group. Nobody was randomised and no treatment was tested, so the study cannot say whether nusinersen or risdiplam helps anyone. Patients were already on treatment before their first assessment, and the group is small and mixed: 27 people could not sit, 25 could sit and only 6 could walk, so the findings may not fit the people you see. The Goal Attainment Scale is individualised by design, so each person was measured against their own goals and the scores cannot be compared between patients. The authors tried to get around this by grouping goals into areas. The two-week reproducibility check covered only 23 of the 58 patients, and the comparison between goal ratings and the motor scales rested on small numbers of people. Data were incomplete for some patients: ventilation status was missing for 12 and scoliosis status for 8, and not every patient had every motor scale recorded. The average gap between the first assessment and follow-up was 10.62 months, but the range ran from 5.60 to 21.08 months, so patients were followed for different lengths of time.

The paper does not say who funded the study. Several authors report personal fees from Biogen, Roche and Novartis outside the submitted work, and one author also reports personal fees from AveXis and Scholar Rock. The remaining authors declare nothing to disclose.

Do not read this as evidence that nusinersen or risdiplam works. Every patient was already on treatment, there was no untreated or comparison group, and the study was about whether patients' own goals can be recorded and tracked, not about whether the drugs changed anything.