Help us amplify the voices of the rare disease community to drive meaningful change and influence policy.
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1 in 10 people
in the U.S. (about 30 million people) live with one or more rare disease.
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$1 trillion
The national economic burden of 379 rare diseases reached
nearly $1 trillion in
the U.S. in 2019. -
~95%
of the more than 10,000 known rare diseases have no FDA-approved therapies.
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15 years
Average time it takes for FDA approval for a rare disease drug.
We Advocate For:
See all issues-
Protecting Medicaid coverage, since cuts to eligibility, benefits, or access would directly harm the rare disease community's ability to get care.
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Join the fight to protect and advance one of America’s most successful public health programs.
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Ensuring the needs of the rare disease community are included in the reauthorization of FDA user fees.
Latest Updates
Take Action
Please Restore the Federal Newborn Screening System
Call on Congress to renew their strong support for evidence-based, federal newborn screening and restore the program.
Events
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RARE Opportunities
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Learn how you can join a community of advocates ages 16-30
Young Adults
Learn how you can join a community of advocates ages 16-30
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Sharing your story through art can help drive meaningful change
Rare Artist
Sharing your story through art can help drive meaningful change
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Connect with other rare disease stakeholders to help determine policy priorities
Community Congress
Connect with other rare disease stakeholders to help determine policy priorities
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