NGLY1 Deficiency clinical trials at UCSF

1 research study open to eligible people

NGLY1 deficiency is a genetic disorder that disrupts protein breakdown in the body. At UCSF, researchers are testing GS-100, a gene therapy delivered directly to the brain. The trial is designed to determine the safety and effectiveness of varying doses.

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  • GS-100 Gene Therapy in Patients With NGLY1 Deficiency

    open to eligible people ages 2-18

    A non-randomized, open-label, dose escalation study of a single intracerebroventricular (ICV) administration of a gene replacement therapy in subjects who are 2 to 18 years old with NGLY1 Deficiency.

    Oakland, California and other locations

Our lead scientists for NGLY1 Deficiency research studies include .

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