NGLY1 Deficiency clinical trials at University of California Health

1 research study open to eligible people

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.
at UCSF

Last updated: March 20, 2025