No single cut-off number has been agreed upon for which a disease is considered rare. With only three diagnosed patients in 27 years, ribose-5-phosphate isomerase deficiency is considered the rarest known genetic disease. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthday. Most rare diseases are genetic and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Orphan drugs are ones so created or sold.
In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments. Disease affecting a small percentage of the populationĪ rare disease is any disease that affects a small percentage of the population.
