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The compound methyl-β-cyclodextrin turns on an enzyme, AMPK, triggering a response against Niemann-Pick type C1, a rare genetic disease

Rare Disease Clues in Cell Recycling System

NCATS scientists discover how drug activates enzyme that reduces cholesterol and fats in the brains and livers of Niemann-Pick Type C1 patients.

ISS with sun rising over Earth

NCATS Supports Tissue Chip Development for Study in Space

NCATS issued five grants to further refine tissue- and organ-on-chip platforms during experiments in-flight to the International Space Station U.S. National Laboratory.

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June E-Newsletter Now Available

Read about NCATS' work to repurpose a drug to treat Duchenne muscular dystrophy, fund studies on tissue chips in space, support a CTSA Program-Eli Lilly training initiative, and more.

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CTSA Program Hubs Collaborate to Enhance Newborn Screening

Efforts are underway to study newborn screening for fragile X syndrome and other genetic diseases.

Duchenne molecule

Potential Drug to Treat Muscular Dystrophy

NCATS researchers and collaborators demonstrated that an unsuccessful cancer drug may have new life as a potential treatment for Duchenne Muscular Dystrophy.

More Center News

Mentorship Advances MS Research

Community Mentorship Helps Advance Multiple Sclerosis Research in Latinos

Trial Innovation Network

CTSA Program-supported investigators tackling inefficiencies in clinical trials

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Work with Us

Learn more about all the ways that potential grantees, partners and collaborators can access NCATS support to advance translational science.

  • NCATS Programs & Initiatives
    NCATS encourages partnerships that cross many scientific disciplines and research sectors in pursuit of translational success.
  • Small Business Opportunities
    NCATS’ small business programs help entrepreneurs develop and commercialize new translational technologies.
  • Expertise & Resources
    NCATS staff are experts in translation, and the Center’s resources support pre-clinical and clinical activities.
  • Licensing Opportunities
    NCATS makes it easy for industry and academia to interact and partner with Center laboratories and scientists.