- Rare Disease: Any disease, disorder, illness or condition affecting fewer than 200,000 people in the United States is considered rare. 1
- There are approximately 7,000 rare diseases. 2
- Most rare diseases are genetic or have a genetic component. 3
- It’s estimated that 25-30 million Americans (almost 1 in 10) have rare diseases. 7
- More than 90% of rare diseases are without an FDA-approved treatment. 9
- For many rare diseases, signs may be observed at birth or in childhood. 5
- Many rare diseases result in premature deaths of infants and young children, or are fatal in early childhood. 4
- There are more than 500 types of rare cancers. 6
- All pediatric cancers are rare. 8
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