186P Demographic and clinical characteristics of risdiplam-treated and untreated adult patients with SMA.

Risdiplam (EVRYSDI®) is a disease-modifying therapy approved for the treatment of spinal muscular atrophy (SMA). The efficacy of risdiplam has been demonstrated in clinical trials that included adult patients, but the real-world effectiveness of risdiplam in adults has not yet been extensively investigated. Natural history studies have shown a slow, progressive decline in motor function in adult patients with SMA. It remains unclear how treatment with risdiplam may influence this trajectory in a real-world clinical setting. As such, this study aims to describe adult patients with SMA treated with risdiplam in terms of their demographic and clinical characteristics, treatment patterns and motor function outcomes, and explore, if possible, the comparison of these outcomes with untreated patients. This non-interventional, retrospective cohort study will use data from six registries (clinician and patient reported) from within the TREAT-NMD network (Australia, Belgium, Canada, Czech Republic & Slovakia, Germany & Austria, and the UK & Ireland) to identify adult patients with SMA treated with risdiplam and where feasible, compare with an untreated patient population. Appropriate adjustment methods will be explored to account for potential confounders where possible, and appropriate statistical models will be used to perform comparisons with untreated patients. As of the submission of this abstract, the analysis is ongoing. Available data will be presented with descriptive statistics. In sum, this study will add to the growing real-world evidence for risdiplam in adults. This study will help to contextualise the effect of risdiplam in this patient population and that of untreated patients. [ABSTRACT FROM AUTHOR]