NGLY1 Deficiency clinical trials at University of California Health

1 in progress, 0 open to eligible people

GS-100 Gene Therapy in Patients With NGLY1 Deficiency
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A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency.
at UCSF

Last updated: April 28, 2026