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On the left is a normal lung and on the right is a lung affected by lymphangioleiomyomatosis.

First Treatment for Rare Lung Disease Approved

NCATS-supported research collaboration leads to FDA approval of drug to treat lymphangioleiomyomatosis.


Rare Diseases Clinical Research Network (RDCRN)

The RDCRN is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.

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About the RDCRN

Learn more about the RDCRN program and its network of study sites.

    RDCRN Funding Information

    Find out about funding opportunities, how to apply and how to collaborate.

    Current RDCRN Consortia

    Discover the 22 consortia and their central data management and coordinating center.

      Rare Diseases Health Information

      Access information about rare diseases for patients and researchers.

        RDCRN Overview

        The RDCRN is designed to advance medical research on rare diseases. It facilitates clinical research in rare diseases through support for:

        • Collaborative activities, including multisite longitudinal studies of individuals with rare diseases, and/or clinical trials
        • Training of clinical investigators in rare diseases research
        • Pilot and demonstration projects
        • Uniform data collection protocols
        • Access to information about rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the public

        Learn more about the RDCRN and its goals.