Caregivers and adult patients with Type 2 and non-ambulatory Type 3 spinal muscular atrophy (SMA) value motor function, breathing function and oral administration in the context of SMA treatments.

Caregivers were more willing to make trade-offs to improve motor function and—to a lesser extent—breathing function, while adult patients focused more on avoiding deterioration in breathing function and motor function.

Disutilities generated from the UK population were substantial for SMA disease outcomes and care aspects.