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

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dc.contributor.authorMartínez Vicente, L.
dc.contributor.authorGajate García, V.
dc.contributor.authorGutiérrez Gutiérrez, Gerardo
dc.contributor.authorGuerrero Peral, Á. L.
dc.contributor.authorHorga Hernández, A.
dc.contributor.authorGuerrero Sola, Antonio
dc.contributor.authorÁlvarez Troncoso, J
dc.contributor.authorRestrepo Córdoba, M. A
dc.contributor.authorGoirigolzarri Artaza, J
dc.contributor.authorMartín Albarrán, S
dc.contributor.authorEt. al.
dc.date.accessioned2026-02-20T12:50:16Z
dc.date.available2026-02-20T12:50:16Z
dc.date.issued2025
dc.description.abstractHereditary 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.en
dc.description.filiationUEMspa
dc.description.impact3.1 Q2 JCR 2024
dc.description.impact0.791 Q2 SJR 2024
dc.description.impactNo data IDR 2024
dc.description.sponsorshipSin financiaciónes
dc.identifier.citationMartínez-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
dc.identifier.doi10.1016/j.nrl.2025.501920
dc.identifier.issn0213-4853
dc.identifier.issn1578-1968
dc.identifier.urihttps://hdl.handle.net/11268/16861
dc.language.isoeng
dc.peerreviewedSi
dc.relation.publisherversionhttps://doi.org/10.1016/j.nrl.2025.501920
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internationalen
dc.rights.accessRightsopen access
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subject.otherAlgoritmos
dc.subject.otherAmiloidosis
dc.subject.otherEnfermedades metabólicas
dc.subject.sdgGoal 3: Ensure healthy lives and promote well-being for all at all ages
dc.subject.unescoAlgoritmo
dc.subject.unescoTratamiento médico
dc.subject.unescoInvestigación médica
dc.titleRedefining 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
dc.title.alternativeRedefiniendo el tratamiento de la ATTRv: análisis de los predictores de respuesta a tafamidis y patisirán en una zona no endémica. Propuesta para un nuevo enfoque terapéutico individualizado
dc.typejournal article
dc.type.hasVersionVoR
dspace.entity.typePublication
relation.isAuthorOfPublicationb7557616-3760-45a7-a37d-86eb9a29e434
relation.isAuthorOfPublication.latestForDiscoveryb7557616-3760-45a7-a37d-86eb9a29e434

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