Rare Diseases & Orphan Drugs

Approximately 7,000 rare diseases are known to exist, and approximately 30 new ones are identified each year. Also known as orphan diseases, they are defined as those which affect fewer than 200,000 patients in America. And while it might be true that each rare disease only affects a small number of patients, collectively, millions of lives are depending on the development of innovative cures and medicines.

Press Releases
October 14, 2019
BIO’s President and CEO Jim Greenwood issued the following statement after the Congressional Budget Office released an analysis confirming that H.R. 3 will lead to fewer new medicines for patients.
May 29, 2019
BIO announces the winner of this year’s Henri A. Termeer Biotechnology Visionary Award: Emil D. Kakkis, MD, PhD, President and Chief Executive Officer, Ultragenyx (NASDAQ: RARE) and Founder, EveryLife Foundation for Rare Diseases. The award, which will be presented to Dr. Kakkis on June 5…
December 5, 2019
More than 135 Emerging Biotech Leaders Urge Congress to Reject H.R. 3  “Our continued success depends on maintaining an environment that supports investment in tomorrow’s discoveries.” Washington, DC: December 5, 2019 – In a letter addressed to House Speaker Nancy Pelosi (D-CA) and House…
Patient-Take-Action
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