NGLY1 Deficiency clinical trials at UCSF
1 in progress, 0 open to eligible people
Sorry, not currently recruiting here
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 Alexander Fay, MD.