Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project.
Irene Serrano-GonzaloLaura López de FrutosCarlos Lahoz-GilFrancisco Delgado-MateosMaría Ángeles Fernández-GalánMontserrat Morales-ConejoMaría Victoria Calle-GordoDaiana Ibarretxe-GerediagaAndrés Madinaveitia-OchoaAntonio Albarracin-ArraigosaJosé Balanzat-MuñozPatricia Correcher-MedinaLuis Javier García-FradeJesús María Hernández-RivasFrancesca LabbadiaJesus Miguel López-DuplaMaría Luisa Lozano-AlmelaElvira Mora-CasteráMaría Soledad Noya-PereiraMaría Ángeles Ruíz-GuinaldoMaría Del Mar Tormo-DíazIsidro Vitoria-MiñanaIsidro Arévalo-VargasMarcio Andrade-CamposPilar GiraldoPublished in: Orphanet journal of rare diseases (2023)
In summary, this real-life study, showed that eliglustat maintains stability and can improve quality of life with few side effects. Significant reductions in classic and other novel biomarkers were observed after two years of therapy.