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Researchers track rare brain disease in children to pave way for future treatments

NCT ID NCT05659901

Summary

This study aims to better understand how Pelizaeus-Merzbacher disease (PMD) progresses over time in children. Researchers will collect spinal fluid, blood samples, and brain scans from up to 32 boys with PMD over about two years. The information gathered will help identify biological markers that could guide the development of future treatments for this rare genetic disorder.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Ionis Investigative Site

    RECRUITING

    Atlanta, Georgia, 30342, United States

  • Ionis Investigative Site

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Ionis Investigative Site

    RECRUITING

    Clermont-Ferrand, France

  • Ionis Investigative Site

    RECRUITING

    Le Kremlin-Bicêtre, 94270, France

  • Ionis Investigative Site

    ACTIVE_NOT_RECRUITING

    Göttingen, Germany

  • Ionis Investigative Site

    RECRUITING

    Tel Aviv, 6423906, Israel

  • Ionis Investigative Site

    RECRUITING

    Milan, 20154, Italy

  • Ionis Investigative Site

    RECRUITING

    Amsterdam, 1081 HV, Netherlands

Conditions

Explore the condition pages connected to this study.