ABSTRACT
The vision is the cure of rare genetic diseases: it is unclear to us why anyone should be disadvantaged just for having inherited a rare genetic disorder. Given the large number of rare diseases, we wanted to avoid spreading our effort thinly across several diseases and having no impact. The chapter also focuses on one condition to try to make a significant difference, and then to broaden our work by applying what we had learned, initially to related conditions. The Myrovlytis Trust aimed to be a research hub, acting as a central focus for the formation of a collaborative global Birt-Hogg-Dub+¬ community. An international organisation that is seen to be disinterested and not competing directly with any other stake-holder can play the primary driving role in creating a community. This applies to the field of rare diseases, but catalysing collaboration in this area faces specific challenges caused by the nature of a tightly focused research area.
