TY  - JOUR
AU  - Vázquez-Costa, Juan F
AU  - Branas-Pampillón, María
AU  - Medina-Cantillo, Julita
AU  - Povedano, Mónica
AU  - Pitarch-Castellano, Inmaculada
AU  - López-Lobato, Mercedes
AU  - Fernández-Ramos, Joaquín A
AU  - Lafuente-Hidalgo, Miguel
AU  - Rojas-García, Ricard
AU  - Caballero-Caballero, José M
AU  - Málaga, Ignacio
AU  - Eirís-Puñal, Jesús
AU  - De Lemus, Mencía
AU  - Cattinari, María G
AU  - Cabello-Moruno, Rosana
AU  - Díaz-Abós, Paola
AU  - Sánchez-Menéndez, Victoria
AU  - Rebollo, Pablo
AU  - Maurino, Jorge
AU  - Madruga-Garrido, Marcos
PY  - 2022
DO  - 10.1007/s40120-022-00411-2
SN  - 2193-8253
UR  - http://hdl.handle.net/10668/21519
T2  - Neurology and therapy
AB  - 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 existing questionnaires and...
LA  - en
KW  - Disease burden
KW  - Outcome measures
KW  - Patients and caregivers
KW  - Quality of life
KW  - Spinal muscular atrophy
KW  - Symptom assessment
TI  - Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study.
TY  - research article
VL  - 12
ER  -