Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe 16/11/2018 Artículos
How to improve specific databases for clinical data in rare diseases? The example of hereditary haemorrhagic telangiectasia 16/11/2018 Artículos
Impact of measures to enhance the value of observational surveys in rare diseases: the Fabry Outcome Survey (FOS) 16/11/2018 Artículos
Inherited arrhythmias: a National Heart, Lung, and Blood Institute and Office of Rare Diseases workshop consensus report about the diagnosis, phenotyping, molecular mechanisms, and therapeutic approaches for primary cardiomyopathies of gene mutations affecting ion channel function 16/11/2018 Artículos
Innovative work behavior in healthcare: the benefit of operational guidelines in the treatment of rare diseases 16/11/2018 Artículos
Hepatocanalicular transport defects: pathophysiologic mechanisms of rare diseases 16/11/2018 Artículos
