NGLY1 Deficiency clinical trials at UCSF

1 research study open to eligible people

NGLY1 deficiency is a rare inherited disorder that affects protein recycling in cells. UCSF is doing a dose escalation trial that gives gene replacement into the brain fluid to evaluate safety and dose. UCSF is also gathering health exams and lab data from participants over time.

Showing trials for

  • 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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