Redefining therapeutics in vATTR: Evaluation of response predictors to tafamidis and patisiran treatment in a non-endemic area. A proposal for a novel individualised therapeutic approach
Loading...
Identifiers
Publication date
Authors
Martínez Vicente, L.
Gajate García, V.
Guerrero Peral, Á. L.
Horga Hernández, A.
Advisors
Editors
Journal Title
Journal ISSN
Volume Title
Publisher
SDG
Metrics
Abstract
Hereditary transthyretin amyloidosis (vATTR) is a progressive genetic disorder with several approved treatments. This study aims to investigate treatment responses to tafamidis and patisiran in vATTR patients to identify predictive response factors. Retrospective analysis on vATTR patients treated with tafamidis or patisiran from October 2012 to September 2022. Treatment responses were assessed as “good,” “partial,” or “non-response.” It was analysed pre-treatment clinical and laboratory data to identify predictors of treatment response.
Description
UNESCO Subjects
Keywords
Bibliographic reference
Martínez-Vicente, L., Gajate-García, V., Gutiérrez-Gutiérrez, G., Guerrero-Peral, Á. L., Horga, A., Guerrero-Sola, A., Álvarez-Troncoso, J., Restrepo-Córdoba, M. A., Goirigolzarri-Artaza, J., Martín-Albarrán, S., Valverde-Megías, A., González-López, E., García-Pavía, P., Matías-Guiu, J., & Galán-Dávila, L. (2025). Redefining therapeutics in vATTR: Evaluation of response predictors to tafamidis and patisiran treatment in a non-endemic area. A proposal for a novel individualised therapeutic approach. Neurología, 501920. https://doi.org/10.1016/j.nrl.2025.501920
Editor's version
https://doi.org/10.1016/j.nrl.2025.501920Type of document
Collections
La licencia de este ítem se describe como Attribution-NonCommercial-NoDerivatives 4.0 International