Rare Disease

A rare disease, also called an orphan disease, is a condition that affects a very small percentage of the population—typically fewer than 200,000 people in the United States. These diseases are often complex, chronic, and life-threatening, and they may involve genetic, infectious, or environmental causes. Due to their rarity, they can be challenging to diagnose and may lack effective treatments. Awareness and research are limited, making support networks crucial for patients and families affected by these conditions. Despite their low prevalence, rare diseases collectively impact millions worldwide.