Browsing by Subject "Malalties rares"
Now showing items 1-3 of 3
-
Análisis de la evolución en el acceso a los medicamentos huérfanos en España
(Gaceta Sanitaria, 2020)Objetivo: Evaluar el acceso al mercado de los medicamentos huérfanos en España que a fecha de 31 de diciembre de 2017 tuvieran vigente su designación, y para aquellos comercializados en España estimar ... -
Gender differences in hereditary hemorrhagic telangiectasia severity
(Orphanet Journal of Rare Diseases, 2020)Background: Gender differences in organ involvement and clinical severity have been poorly described in hereditary hemorrhagic telangiectasia (HHT). The aim of this study was to describe differences in ... -
Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper
(Orphanet Journal of Rare Diseases, 2023)Background: In recent years, significant advances have been made in the field of rare diseases (RDs). However, there is a large number of RDs without specific treatment and half of these treatments have ...