Because Every Person With a Disease Deserves Treatment Options

Join our mission to protect bipartisan incentives that have made rare disease therapies a reality.

The ORPHAN Cures Act

Bipartisan, federal legislation to protect research into treatments for the millions of Americans currently suffering from rare diseases and rare cancers.

Rare Disease at-a-Glance

Get a quick glimpse into the staggering numbers that define the world of rare diseases and orphan drugs.
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Rare diseases

There are approximately 10,000 different rare diseases.

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have an approved treatment

Less than 10% of rare diseases have an FDA approved treatment available.

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Americans

Nearly 30 million people in the U.S. have a rare disease.

Watch: The Importance of Protecting Rare Disease Innovation

Learn about the ORPHAN Cures Act, a crucial bipartisan initiative designed to support innovation in rare disease treatments. Hear from experts and advocates Paul Kim, Arya Singh, and Lisa Schlager as they explain why this legislation is vital to encouraging drug innovation, expanding treatment options, and offering hope to rare disease patients.