Psychometric evaluation of modified spinal muscular atrophy functional rating scale (SMAFRS) in adult patients using Rasch analysis.
Reza SadjadiKristina KellyAllan M GlanzmanJacqueline MontesMatthew LinsenmayerMarco TellezAmy BartlettSarah HeintzmanStephen J KolbW David ArnoldBakri El-SheikhPublished in: Muscle & nerve (2023)
Despite subtle misfitting of certain items, mainly related to respiratory and bulbar function, overall modified SMAFRS remained a psychometrically stable and unidimensional outcome measure. There were some differences in measuring properties of certain functional items between ambulatory and non-ambulatory items that need to be taken into consideration in clinical trial design. Overall, the modified SMAFRS is a psychometrically reliable tool in assessment of adult patients with SMA.