Collaboration: A key to unlock the challenges of rare diseases research
Rare diseases present unique research challenges. The small number of people affected by a rare disease, each with its specificities combined with the frequent lack of understanding about the cause and course of the diseases, leads to a scarcity and a fragmentation of knowledge and expertise. People living with a rare disease often need highly specialised healthcare and social services, resulting in time lost to finding the right services and high costs for their families and for society.