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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Ngā kaituhi matua:	Maria Grazia Cattinari, Samuel Ignacio Pascual-Pascual, Mencía de Lemus, Julita Medina, María Dumont, Pablo Rebollo, Juan Francisco Vázquez-Costa
Hōputu:	Artigo
Reo:	Inglês
I whakaputaina:	BMC 2025-06-01
Rangatū:	Orphanet Journal of Rare Diseases
Ngā marau:	Spinal muscular atrophy Patient reported outcomes Psychometric properties Quality of life Treatment outcomes
Urunga tuihono:	https://doi.org/10.1186/s13023-025-03832-y
Ngā Tūtohu:	Tāpirihia he Tūtohu Kāore He Tūtohu, Me noho koe te mea tuatahi ki te tūtohu i tēnei pūkete!