NGLY1 Deficiency clinical trials at University of California Health
1 research study open to eligible people
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.
at UCSF
Our lead scientists for NGLY1 Deficiency research studies include Alexander Fay, MD.
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