Collectively, rare diseases affect one in ten Americans

WASHINGTON, D.C. (October 7, 2013)

America’s biopharmaceutical research companies are developing 452 new medicines for rare diseases, including genetic disorders, neurological conditions, infectious diseases and autoimmune disorders, according to a new report released today by the Pharmaceutical Research and Manufacturers of America (PhRMA).

Biopharmaceutical research is entering an exciting new era with a growing understanding of the human genome. Scientific advances have given researchers new tools to explore rare diseases, which are often more complex than common diseases. According to the Food and Drug Administration (FDA), in the last five years, one third of all new drug approvals were for rare diseases. Read more