Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.

Background: Novel Spinal Muscular Atrophy (SMA) treatments have demonstrated improvements on motor measures that are clearly distinct from the natural history of progressive decline. Comparable measures are needed to monitor bulbar function, which is affected in severe SMA.

Objective: To assess bulbar function with patient-reported outcome measures (PROs) and determine their relationships with clinical characteristics.