Working for patients with rare, low-prevalence and complex diseases

Working for patients with rare, low-prevalence and complex diseases

ERN on immunodeficiency, autoinflammatory and autoimmune diseases (ERN RITA) RITA brings together the ERN RITA is working to reduce leading European centres inequalities faced by patients with expertise in diagseeking to access diagnostic nosis and treatment of rare immunological distesting and innovative orders. These constitute treatments. potentially life-threatening conditions requiring multidisciplinary care using complex diagnostic evaluation and highly specialised therERN RITA is working to reduce inequalities apies. The network divides these conditions faced by patients seeking to access diagnostic into three sub-themes: primary immunodetesting and innovative treatments such as bioficiency (PID), autoimmune disorders and logic therapies, immunoglobulin replacement, autoinflammatory disorders. In addition, there stem cell transplantation and gene therapy. is a sub-theme of paediatric rheumatology which straddles the autoimmune and autoIt aims to link pre-existent registries, develop inflammatory sub-themes. pan-European clinical guidelines, establish a task force of geneticists for quality control This network builds on the work of European of next generation sequencing technology, scientific societies which have developed agree a common tool for pharmacovigilance patient registries, clinical guidelines, research in these rare conditions, convene a task force collaborations, educational activities and links for the correct use and monitoring of biologic with patient organisations. treatments in immune-mediated diseases,

bring together and improve stem cell and gene therapies for patients, foster collaborations between patient associations, and bring together paediatric and adult specialists across the three themes.

NETWORK COORDINATOR Professor Andrew Cant The Newcastle upon Tyne Hospitals NHS Foundation Trust, United Kingdom