Gaucher Disease clinical trials at UCSD

1 research study open to eligible people

Showing trials for

  • Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

    open to eligible people ages 18-50

    This is a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and postpubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).

    San Diego, California and other locations

Our lead scientists for Gaucher Disease research studies include .

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