The conference sessions will explore issues and cutting-edge technologies that affect many adult and paediatric conditions. The following topics are to be discussed:Stem cell and cell therapy approachesGenetic mapping and genomic instabilityPathophysiology and diagnosticsDegenerative disordersRegulatory affairs and patients in researchOur aim is to promote research on rare and orphan diseases among the general public, industry and policymakers as well as to provide a forum for researchers to meet and pool their knowledge. The underlying objective is to tackle the key issues that need to be addressed if we are to see new and promising therapies and treatments rapidly delivered to patients all around the world.