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U.S. Department of Health and Human Services

For Industry

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Videos: Patients with Rare Diseases and Conditions

About 30 million Americans are affected with a rare disease, for which there may be few or no treatments. A rare disease is one that affects fewer than 200,000 people.

Through the Orphan Drug Act and other legislation, FDA provides incentives to industry to help develop treatments for rare diseases. The agency also advocates for rare disease awareness and support through work with patients and patient organizations.

Each of the 7,000 known rare diseases has unique challenges, which may have  a significant impact on patients with rare diseases and their families on a daily basis.  

Although they face challenges, many patients speak out with strong voices to raise awareness of their debilitating diseases. Some of their stories are shared here.

Duchenne Muscular Dystrophy: Charlie's Story

Isovaleric Acidemia: Stephen and Caroline's Stories



For slideshow captions, click the play button in the center, the full screen icon in the lower right corner, then "Show Info" in the upper right corner.