RecruitingPHASE1, PHASE2NCT06199531
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
Studying Alacrimia-choreoathetosis-liver dysfunction syndrome
Last synced from ClinicalTrials.gov
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- Grace Science, LLC
- Intervention
- GS-100(genetic)
- Enrollment
- 6 enrolled
- Eligibility
- 2-18 years · All sexes
- Timeline
- 2024 – 2028
Study locations (3)
- Oakland Children's Hospital (UCSF Benioff), Oakland, California, United States
- Columbia University Irving Medical Center, New York, New York, United States
- Texas Children's Hospital (Baylor College of Medicine), Houston, Texas, United States
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
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