Post hoc analysis of the SMA independence scale-upper limb module using SUNFISH Part 2 data

<h3></h3> The SMA Independence Scale-Upper Limb Module (SMAIS-ULM) is a validated clinical assessment, designed to assess the level of support required for activities of daily living (ADLs) related to upper limb function (ULF) in individuals with Type 2 and non- ambulant Type 3 SMA. In the SUNFISH trial Part 2 (NCT02908685), both patient-reported (n=68; ≥12–25 years) and caregiver-reported (n=180; 2–25 years) SMAIS-ULM versions were analysed. This post hoc analysis investigates the impact of risdiplam on ULF and levels of independence in ADLs across different age groups. During the placebo-controlled period at Month 12, the mean total scores in patient-reported SMAIS-ULM displayed a numerical increase from baseline in the risdiplam age subgroups (≥12–25 years) in contrast to a decline in the placebo age subgroups. Caregiver-reported SMAIS-ULM also exhibited a numerical increase favouring risdiplam across age subgroups, excluding the 18–25 years group. Responder analyses indicated a larger proportion of risdiplam-treated individuals showing improvement or stabilisation in SMAIS-ULM total score from baseline, compared to placebo across all age subgroups for both patient- and caregiver-reported total scores. The current findings highlight the benefit of risdiplam on ULF, and levels of independence in ADLs, across children, teenagers, and adults with more advanced disease.