5^th International Conference on Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs: Rare diseases are medical conditions that affect a relatively small proportion of the population and may involve complex symptoms, genetic changes, or abnormalities in specific organs. Orphan drugs are medicines developed to prevent, diagnose, or treat these conditions, particularly where effective treatment options are limited. This session explores disease mechanisms, diagnostic challenges, treatment development, patient care, and strategies for improving access to therapies for people living with rare diseases