Ankara NADİR seeks to improve diagnosis and treatment approaches in the field of rare diseases within a multidisciplinary system based on a single umbrella organization model, thus increasing the quality of life of patients, increasing clinical research, balancing supply and demand with the information obtained, carrying out R&D (Phase I, II, III) and devising production plans for orphan drugs used in therapies and advanced medical treatments, and offering academic degrees to prospective scientists who are specialized in this field.

In line with our objectives, we intend to determine the burden stemming from rare diseases in our country, to support the national registry systems to be established for priority diseases, to formulate strategies for prenatal and newborn screenings, to provide patients and carriers with easy access to counselling, to conduct R&D projects for the creation of early diagnosis and treatment kits, approaches, etc., to create diagnostic algorithms for diseases, to define treatment models and to prepare treatment guides.

In addition to gathering scientific data, we aim also to ensure the efficient use of the garnered information by raising public awareness on the diagnosis and treatment of rare diseases, and to contribute to the development of policies on rare disease through pharmacoeconomic studies.