The Foundation’s mission is to encourage therapeutic research for rare and orphan diseases.
Its goals are:
- To collect funds directed toward research in rare and orphan diseases.
- To support translational and clinical research by allocating resources to investigators working on rare and orphan diseases.
- To collect funds directed toward research for a specific rare and orphan diseases (e.g. liver metabolic diseases).
- To promote and encourage therapeutic trials aimed at developing curative drugs for rare and orphan diseases.
- To inform the public on rare and orphan diseases.