NGLY1 Deficiency clinical trials at UCSF

1 research study open to eligible people

NGLY1 deficiency is caused by the absence of an essential enzyme in the body. UCSF trials involve applying gene therapy directly into the brain's fluid to evaluate patient reactions. Researchers adjust doses to see how safety and effectiveness change.

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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