A rare disease typically affects fewer than five in 10,000 people worldwide. Despite the rarity of each disease individually, when considered collectively, rare diseases affect a significant population – there are roughly 30 million people in the US and more than 300 million people worldwide living with a rare disease. There are nearly 7,000 different rare diseases currently identified, while only about 5% have an FDA-approved treatment. In addition, these diseases are often debilitating and potentially fatal; about 30% of children with a rare disease will die before their fifth birthday. Approximately 80% of rare diseases have identified genetic origins.