One-Shot gene therapy trial offers new hope for rare disease

NCT ID NCT06539624

Summary

This study is testing a new, one-time gene therapy called EXG110 for people with Fabry disease, a rare genetic disorder. Researchers want to find a safe dose and see if it can help control the disease by improving kidney and heart function. The study is enrolling a small group of adults and children aged 7 and older.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Children's Hospital, Zhejiang University School of Medicine

    RECRUITING

    Hangzhou, Zhejiang, China

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

  • Shanghai Children's Medical Center

    RECRUITING

    Shanghai, Shanghai Municipality, China

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

    Contact

Conditions

Explore the condition pages connected to this study.