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

Autor: Juan F. Vázquez-Costa, María Branas-Pampillón, Julita Medina-Cantillo, Mónica Povedano, Inmaculada Pitarch-Castellano, Mercedes López-Lobato, Joaquín A. Fernández-Ramos, Miguel Lafuente-Hidalgo, Ricard Rojas-García, José M. Caballero-Caballero, Ignacio Málaga, Jesús Eirís-Puñal, Mencía De Lemus, María G. Cattinari, Rosana Cabello-Moruno, Paola Díaz-Abós, Victoria Sánchez-Menéndez, Pablo Rebollo, Jorge Maurino, Marcos Madruga-Garrido

Publikováno v: Neurology and Therapy, Vol 12, Iss 1, Pp 89-105 (2022)

Abstract Introduction Outcome measures traditionally used in spinal muscular atrophy (SMA) clinical trials are inadequate to assess the full range of disease severity. The aim of this study was to assess the psychometric properties of a set of existi

Externí odkaz: https://doaj.org/article/9bfcddc000ee4147b49cc5667317fe56

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Design of a Non-Interventional Study to Validate a Set of Patient- and Caregiver-Oriented Measurements to Assess Health Outcomes in Spinal Muscular Atrophy (SMA-TOOL Study)

Autor: Madruga-Garrido M, Vázquez-Costa JF, Medina J, Brañas M, Cattinari MG, de Lemus M, Díaz-Abós P, Sánchez-Menéndez V, Terrancle Á, Rebollo P, Maurino J

Publikováno v: Neurology And Therapy
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
Neurology and Therapy
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu

INTRODUCTION: There is a need to optimize the current clinical outcome measures in spinal muscular atrophy (SMA) incorporating patients' and caregivers' perspectives. The aim of this study is to evaluate the psychometric properties (validity, reliabi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::d322fd25b0cc20cf7015fc6dcd551c9b
https://fundanet.iislafe.san.gva.es/publicaciones/ProdCientif/PublicacionFrw.aspx?id=13726

VP.56 Perception of treatment efficacy among pediatric neurologists caring for patients with spinal muscular atrophy

Autor: Saposnik, G., Camacho, A., Díaz-Abós, P., Brañas, M., Sánchez-Menéndez, V., Cabello-Moruno, R., Terzaghi, M., Mauriño, J., Malaga, I.

Publikováno v: In Neuromuscular Disorders October 2022 32 Supplement 1:S92-S92