Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol.

Pablo RebolloSofía García-LópezMónica PovedanoMaría G CattinariMercedes Martínez-MorenoÁngeles TerrancleRosana Cabello-MorunoJuan Francisco Vázquez Costa

Published in: Neurology and therapy (2024)

The validation of this tool will facilitate the clinical evaluation of adult and adolescent patients with SMA and the quantification of their response to new treatments in both clinical practice and research.

Keyphrases

young adults
clinical practice
study protocol
randomized controlled trial
childhood cancer
mental health
physical activity
clinical trial
open label