Preliminary psychometric validation of patient-reported outcomes relevant to individuals with spinal muscular atrophy and their caregivers

Abstract Background There is a need to expand the current scope of assessment tools usually applied to patients with Spinal Muscular Atrophy (SMA). This study aimed to assess the psychometric properties (reliability and discriminant validity) of a set of new patient-reported outcome measures (PROMs)...

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Bibliographic Details
Main Authors: Maria Grazia Cattinari, Samuel Ignacio Pascual-Pascual, Mencía de Lemus, Julita Medina, María Dumont, Pablo Rebollo, Juan Francisco Vázquez-Costa
Format: Article
Language:English
Published: BMC 2025-06-01
Series:Orphanet Journal of Rare Diseases
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Online Access:https://doi.org/10.1186/s13023-025-03832-y
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