Biomedicine & Pharmacotherapy

Rare Disease Therapeutics

Rare Disease Therapeutics

Rare diseases are conditions that affect a small percentage of the population, typically defined as diseases that impact fewer than 200,000 people in a given population (such as in the U.S.). Despite their rarity, these diseases often present significant challenges due to their complexity, the limited number of effective treatments, and the lack of awareness in the medical community. As a result, rare disease therapeutics focuses on the development, approval, and access to treatments for these uncommon and often neglected conditions.