Biomedicine & Pharmacotherapy

Rare and Orphan Diseases: Therapeutic Approaches

Rare and Orphan Diseases: Therapeutic Approaches

Rare diseases are conditions that affect a small percentage of the population. The definition varies by region, but in the U.S., a rare disease affects fewer than 200,000 people, while in the EU, it affects fewer than 1 in 2,000 individuals. Orphan diseases are rare conditions for which there are limited or no effective treatments.