Patient demographics and clinical characteristics of adults with SMA: assessment of risdiplam-treated and untreated SMA

<h3></h3> 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 This non-interventional, retrospective cohort study aims to describe adult SMA patients in terms of their demographic and clinical characteristics, treatment patterns and healthcare resource utilisation. If possible, it will also explore how newly treated SMA adults receiving risdiplam compare with patients who are not receiving DMTs. This study will use data from seven registries (clinician and patient reported) from within the TREAT-NMD network (Australia, Belgium, Canada, Czech Republic &amp; Slovakia, Germany &amp; Austria, Ukraine, UK and Ireland) to identify adult SMA patients treated with risdiplam and compare with an untreated patient population, in registries deemed feasible for a comparative analysis. Appropriate adjustment methods will be explored to adjust for potential confounders, and appropriate statistical models will be used to perform comparisons with untreated patients. The analysis of this study is ongoing as of the submission of this abstract. Available data from the comparative analyses will be presented. This study will provide additional evidence to further support the understanding of treatment effectiveness in adults with SMA.