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A 2017 Rare Disease Day at NIH exhibitor speaks to an event participant.

Scientist-Patient Advocate Team Advances NPC1 Research

Patient-focused collaboration among government and academic scientists enabled the advance of a potential treatment for the rare disease Niemann-Pick type C1.

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Discover NCATS' collection of featured research stories and scientific publications, or browse the latest issue of NCATS' monthly e-newsletter.

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        NCATS in the News

        NCATS-supported scientists are engaged in cutting-edge translational research activities across the United States. See breaking news and media coverage of the latest advances made possible by support from the Center.