National Registries of Rare Diseases in Europe: An Overview of the Current Situation and Experiences 16/11/2018 Artículos
New and evolving rare diseases research programs at the National Institutes of Health 16/11/2018 Artículos
Management options for rare diseases in children and adolescents in Georgia (experience of the country with transitional economy) 16/11/2018 Artículos
Meeting summary: Ethical aspects of whole exome and whole genome sequencing studies (WES/WGS) in rare diseases, Tel Aviv, Israel, January 2013 16/11/2018 Artículos
Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry 16/11/2018 Artículos
Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates 16/11/2018 Artículos
Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy 16/11/2018 Artículos
