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NCT04532047
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +39 more
- Locations
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- University of California San Francisco, California, United States
NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
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- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
Active, Not Recruiting
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
NCT07236606
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +24 more
- Locations
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- St. Peter's University Hospital New Brunswick, New Jersey, United States