Rare diseases and orphan drugs.
Because every life counts.
A rare disease is any disease that affects a small or very small percentage of the population. It is estimated that more than 300 million people worldwide are living with one of the 7,000 diseases they define as rare. A disease may be considered rare in one part of the world, or in a particular group of people, but still be common in another. Most rare diseases are genetic, and thus are present throughout the person’s entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday. With the constant evolution of medical research, it is possible to imagine a world where all diseases, common or rare have a cure.