Scientists track rare muscle disease to pave way for future treatments

NCT ID NCT01417533

Summary

This study aims to learn more about GNE myopathy, a rare genetic disease that causes progressive muscle weakness starting in young adulthood. Researchers will observe up to 125 participants over time to understand how the disease progresses, what symptoms occur, and to find markers that could help test future treatments. No treatment is provided as part of this study; it is purely for observation and data collection.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.