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Rare Diseases



There are approximately 7,000 rare diseases and treatments exist for less than 500 of them.  We can do better than this.  We must do better than this. 

People living with rare diseases have incredible courage.  They have to, because they have few, if any, treatment options and are often over looked because of the rarity of their disorders. 

At NPS Pharma our mission is to pioneer and deliver innovative therapies that transform the lives of patients with rare diseases worldwide. Our sole focus is rare diseases.  That’s all we do. 

By definition, a rare disease is one that affects less than 200,000 people in the U.S. and up to 245,000 in Europe. In the U.S. and Europe, an estimated 60 million people are living with a rare disease -- this is approximately 10 percent of the U.S. population and six percent of the European population. 

Many rare diseases are severe with life-threatening consequences.  They have a profound impact on patients, families and communities, often making it challenging for people to work, care for their families and live independently.  Rare diseases present unique public health challenges: Compared to more common diseases, there are very few researchers and companies exploring rare diseases and very few physicians who have experience diagnosing and treating these disorders.