Rare Diseases: The Challenges and Rewards of Orphan Drug Development
Rare diseases, also known as orphan diseases, affect a small percentage of the population, typically fewer than 200,000 individuals in the United States. Despite their rarity, these diseases collectively impact millions of people worldwide. Developing treatments for rare diseases presents unique challenges, but it also offers significant rewards for pharmaceutical companies, researchers, and, most importantly, patients. The field of Life Sciences Research plays a crucial role in advancing orphan drug development, overcoming scientific and economic barriers, and bringing hope to those affected by rare conditions.