Rare Diseases

Rare Disease: Any disease, disorder, illness or condition affecting fewer than 200,000 people in the United States is considered rare.

There are approximately 10,000 rare diseases.

Most rare diseases are genetic or have a genetic component.

It’s estimated that 30 million Americans (almost 1 in 10) have rare diseases.

More than 95% of rare diseases are without an FDA-approved treatment.

For many rare diseases, signs may be observed at birth or in childhood.

Many rare diseases result in premature deaths of infants and young children, or are fatal in early childhood.

There are more than 500 types of rare cancers.

All pediatric cancers are rare.