Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study

202224 citationsJournal Articlegold Open Access

Authors

Juan F. Vázquez‐Costa · Universitat de València

María Brañas-Pampillón · Roche (Spain)

Julita Medina-Cantillo · Hospital Sant Joan de Déu Barcelona

Mónica Povedano · Institut d'Investigació Biomédica de Bellvitge

Inmaculada Pitarch Castellano · Hospital Universitari i Politècnic La Fe

Mercedes López-Lobato · Universidad de Sevilla

Joaquín Alejandro Fernández‐Ramos

Abstract

This set of outcome measures showed adequate reliability, construct validity, and discriminant validity and may constitute a valuable option to measure symptom severity in patients with SMA.

Topics & Keywords

Neurogenetic and Muscular Disorders Research Family and Disability Support Research Cerebral Palsy and Movement Disorders

UN Sustainable Development Goals

Reduced inequalities

Publication Details

Published in: Neurology and Therapy

Volume 12, Issue 1, pp. 89-105

DOI: 10.1007/s40120-022-00411-2

Field-Weighted Citation Impact: 3.37