Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry 16/11/2018 Artículos
Protein replacement therapies for rare diseases: a breeze for regulatory approval? 16/11/2018 Artículos
Rare diseases – avoiding misperceptions and establishing realities: the need for reliable epidemiological data 16/11/2018 Artículos
Primary biliary cirrhosis and hereditary hemorrhagic telangiectasia: When two rare diseases coexist 16/11/2018 Artículos
Prioritizing treatment of rare diseases: a survey of preferences of Norwegian doctors 16/11/2018 Artículos
