Rare diseases

In the United States, a rare disease is defined as any disorder affecting fewer than 200,000 people. According to the National Organization for Rare Disorders, 10% of Americans live with one of 10,000 known rare diseases, yet only 5% have an FDA-approved treatment. Those living with rare diseases face medical, emotional, and social impact which can be lifelong and extend to entire families, underscoring the importance of improved diagnosis, coordinated care, continued research, education, and policies that support patients and caregivers.