The EU legal framework for medicines for human use is intended to ensure a high level of public health protection and to promote the functioning of the internal market, and includes measures that encourage innovation.
The Romanian Foundation for Lysosomal Diseases (FRBL), founded in 1998 in Cluj-Napoca, has marked 20 years since its inception.
A Webex aimed to reach potential new CPMS users in MetabERN.
Il progetto è rivolto esclusivamente alle Associazioni di pazienti con malattia rara della Sardegna e promuove interventi di sensibilizzazione, formazione, accompagnamento e comunicazione per favorire l’azione delle organizzazioni dei pazienti e migliorare la situazione dei malati rari nelle regioni del Sud, attraverso un processo di empowerment individuale, organizzativo e di comunità.
Patient-centered research tool to increase knowledge on immune system (dys)function among CDG children and adults - The Immunology and CDG Questionnaire (ImmunoCDGQ) for Patients & Caregivers.
ERN-LUNG is organizing a webinar on how to set up a clinical trials network.
Effective ways of Investing in Health, the Expert Panel will maintain a Hearing on the application of the ERN model in European cross-border healthcare cooperation outside the rare diseases area.
Modern Management of Inherited Metabolic Disorders course hosted by Recordati on in Rio de Janeiro, Brazil 18 - 20 October 2018.
Scientific and technological advancements are rapidly enabling neonatal screening for ever more congenital conditions to become available.
The #IGD2018 #rarestars campaign will focus upon doctors, nurses, scientists, patient advocates and those involved in the pharmaceutical industry, who all work hard to improve the quality of life for the entire Gaucher patient community.