NGLY1 Deficiency clinical trials at UCSF

1 in progress, 0 open to eligible people

GS-100 Gene Therapy in Patients With NGLY1 Deficiency
Sorry, in progress, not accepting new patients
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.
Oakland, California and other locations

Last updated: April 28, 2026