The National Institutes of Health estimates that there are approximately 6,000 rare diseases affecting 25 million Americans. A rare disease is defined as a condition affecting fewer than 200,000 people in the United States. In the past, treatment options for such conditions have been non-existent or limited, but approval of several new medicines in this research sector over the last few years has fostered great progress.

Report:  2007: Orphan Drugs in Development for Rare Diseases (PDF)

Informe: 2007: Medicamentos Huérfanos en Desarrollo para (PDF)

Press Release: More Than 300 Medicines Now in Development to Treat Rare Diseases

Backgrounder: Selected Examples of Orphan Drugs in Development for Rare Diseases