Genetic disorders are caused by abnormalities in genes or chromosomes. They are chronically debilitating, sometimes life-threatening diseases with a low prevalence and a high level of complexity and heterogeneity. There are many high unmet medical needs in the field of genetic disorders, with only few disease-modifying drugs available. These rare disorders call for a global approach to special and combined efforts to prevent significant morbidity or avoidable premature mortality, and to improve the quality of life of those affected.
