NCATS and Rare Diseases Research
Rare Disease Day takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. Since 2011, NCATS has sponsored Rare Disease Day at NIH as part of this global observance. Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect and NIH collaborations that address scientific challenges and advance research for new treatments.
The goals of Rare Disease Day at NIH are to:
- Demonstrate the NIH commitment to advancing research for people affected by rare diseases.
- Highlight NIH-supported rare diseases research and the development of diagnostics and treatments.
- Initiate a mutually beneficial dialogue among the rare diseases community.
- Exchange the latest rare diseases information with stakeholders to advance research and therapeutic efforts.
- Shine a spotlight on stories told by patients living with a rare disease, their caregivers, and their communities.
HSP and PLS are rare neurodegenerative diseases. There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. In the United States, rare diseases affect millions of people. On February 27, 2026, NIH hosted its annual Rare Disease Day event to raise awareness about these disorders, the people they affect, and NIH collaborations that advance research for new treatments. Sponsored by the National Center for Advancing Translational Sciences (NCATS), the event featured panel discussions, rare diseases stories, and more. Listen to the entire meeting
