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New study tests enzyme replacement for rare genetic disease in chinese patients

NCT ID NCT05054387

First seen Oct 31, 2025 · Last updated May 24, 2026 · Updated 29 times

Summary

This study tested the safety and effectiveness of Fabrazyme, an enzyme replacement therapy, in 22 Chinese patients with Fabry disease. Fabry disease is a rare genetic disorder that causes pain, organ damage, and other serious symptoms. Patients received Fabrazyme infusions for 54 weeks, and researchers monitored side effects and changes in disease markers. The goal was to see if this treatment is safe and helps control the disease in this specific population.

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Contacts and locations

Locations

  • Investigational Site Number :1560001

    Shanghai, 200025, China

  • Investigational Site Number :1560002

    Beijing, 100730, China

  • Investigational Site Number :1560003

    Beijing, 100034, China

  • Investigational Site Number :1560004

    Shanghai, 201102, China

  • Investigational Site Number :1560005

    Wuhan, 016040, China

  • Investigational Site Number :1560006

    Taiyuan, 030001, China

Conditions

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