Rare Disease by the Numbers

Rare diseases are surprisingly common.

Rare disease by the numbers:

• Over 7,000 rare diseases exist
• One in 10 Americans is affected by a rare disease
• 80% of rare diseases involve a faulty gene, providing ideal opportunities for gene therapy
• 30 million people in the U.S. and 350 million people worldwide have a rare disease
• 50% of rare diseases affect children, causing death or debilitating illness at a young age
• Rare diseases affect more people than AIDS and cancer-related illness combined
• Only 5% of all rare diseases currently have a treatment or cure