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NCT07054515
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
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- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
NCT07399704
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
- Locations
-
- Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Água Verde, Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT00668187
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- University of Minnesota - Pediatric Genetics and Metabolism Minneapolis, Minnesota, United States
Recruiting
The Myelin Disorders Biorepository Project
NCT03047369
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
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- Children's Hospital of Los Angeles Los Angeles, California, United States
- Children's Hospital of Orange County Orange, California, United States
- Stanford University (Lucile Packard Children's Hospital) Palo Alto, California, United States
- University of California, Davis (UC Davis Health) Sacramento, California, United States
NCT05758922
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT02851862
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Massachusetts General Hospital Boston, Massachusetts, United States
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
NCT03759665
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- University of California - Los Angeles Los Angeles, California, United States
- Mayo Clinic Rochester, Minnesota, United States
- NYU Langone School of Medicine New York, United States
- University of Giessen Giessen, Germany
NCT04470713
- Conditions
- AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
- Locations
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- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Mayo Clinic - Rochester Rochester, Minnesota, United States
- Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
- UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
Active, Not Recruiting
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT06614569
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UMass Chan Medical School Worcester, Massachusetts, United States